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FDA Approves IV Tx for HER2 Breast Cancer – Margenza

December 29, 2020Winter Garden, FL
More catching up —
The FDA approved another specialty therapy earlier this month. The new approval is Margenza (margetuximab-cmkb) from MacroGenics and is indicated for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens. It the first HER2-targeted therapy to have improved progression-free survival (PFS) versus Herceptin (trastuzumab), when both are combined with chemotherapy.
Margenza is an infused therapy. Other 2020 approvals for HER2 breast cancer have been orals.Pricing was not announced at the time of approval.Product launch is targeted for March 2021.
Because of its anticipated high cost and a black box warning, Margenza will likely launch as yet another distribution product via a specialty pharmacy, direct-to-office/hospital.

MacroGenics Announces FDA Approval of Margenza  for Patients with Pretreated Metastatic HER2-Positive Breast Cancer

ROCKVILLE, MD, Dec. 16, 2020 (GLOBE NEWSWIRE) — MacroGenics, Inc., a biopharmaceutical company focused on developing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer, today announced that the U.S. Food and Drug Administration (FDA) has approved Margenza  , in combination with chemotherapy, for the treatment of adult patients with metastatic HER2-positive breast cancer who have received two or more prior anti-HER2 regimens, at least one of which was for metastatic disease. Margenza is the first product approved from MacroGenics’ promising pipeline. The approval was based on safety and efficacy results from the pivotal Phase 3 SOPHIA trial.

“The approval of Margenza…..
Click here to read the full press release
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Executive Editor
William Sullivan

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Jim Larweth
Anton Health


SCOTUS Gift to Specialty Pharmacy Will Boost 2021 Revenues

There really hasn’t been a lot of good news in the specialty pharmacy industry this year. Demand has been down due to the virus. Increasing DIR fees continue to bleed off profits. Virtually no FDA 2020 approvals will come to market in open access. But, the press release below from the National Association of Specialty Pharmacy (NASP) unwraps a gift under the 2020 specialty pharmacy Christmas tree.

Recently, the US Supreme Court issued an 8-0 decision confirming a state’s right to regulate the level of reimbursement by PBMs to specialty pharmacies. It has the effect of ensuring that specialty pharmacies should not be reimbursed at a rate lower than their actual acquisition cost. In short, pharmacies will not have to suffer an automatic loss on many prescriptions where the PBM arbitrarily sets reimbursement in RED levels. That is a BIG deal and the best gift that the industry could hope for at the close to a very challenging year.

Specialty Pharmacy Applauds Supreme Court Decision to Allow States to Regulate PBM Practices

WASHINGTON, Dec. 11, 2020 /PRNewswire/ — The National Association of Specialty Pharmacy (NASP) applauds the 8-0 decision by the Supreme Court to restore Arkansas’ right to govern the amount that Pharmacy Benefit Managers (PBMs) reimburse pharmacies, in Rutledge v. the Pharmaceutical Care Management Association (PCMA). This decision will give states more flexibility to oversee PBM reimbursement practices to ensure patient access and affordability and protect market competition.

Since 2012, NASP members have reported a dramatic decrease in prescription reimbursement and an increase in claw-back fees such as Direct and Indirect Remuneration (DIR) by many PBMs.

“NASP is pleased with the Supreme Court’s unanimous ruling and believes this decision will protect states’ rights to oversee and control abusive practices. Reimbursements from PBMs that are below a specialty pharmacy’s cost threaten patient care and medication access. Specialty pharmacies provide critical care and services for patients living with cancer, rheumatoid arthritis, multiple sclerosis, cystic fibrosis, HIV/AIDS, and other chronic and complex diseases. Today’s decision is a key step in providing the oversight needed to ensure patients have access to the quality care that specialty pharmacies provide,” said Sheila M. Arquette, RPh, NASP President & CEO.”

Contact: Sheila M. Arquette, RPh, President & CEO, (703) 842-0122

The National Association of Specialty Pharmacy (NASP) is the only national association representing all stakeholders in the specialty pharmacy industry. NASP members include the nation’s leading specialty pharmacies, pharmaceutical and biotechnology manufacturers, group purchasing organizations, patient advocacy groups, integrated delivery systems and health plans, technology and data management vendors, wholesalers/distributors, healthcare providers, and practicing pharmacists. With over 130 corporate members and approximately 1,800 individual members, NASP is the unified voice of specialty pharmacy in the United States.

SOURCE National Association of Specialty Pharmacy


Centene Acquires PantherRx Rare Specialty Pharmacy

This year saw few specialty pharmacy acquisitions…. but that just changed with the announcement by Centene Corporation that it has acquired PantherRx Rare Pharmacy. Terms were not disclosed.

It is an important acquisition, but first, let’s look at Centene.
Centene Corporation is a large, publicly-traded, multi-line managed care enterprise that serves as a major intermediary for government-sponsored managed care plans for uninsured, underinsured, and low-income individuals. It also offers traditional, privately-insured health care programs.

Centene also contracts with other healthcare and commercial organizations to provide specialty services, including pharmacy benefits management (PBM), specialty pharmacy dispensing, mail service pharmacy, behavioral health care services, case management software, correctional insurance, in-home health services, life and health management, vision, and telehealth services. (Wikipedia).

Centene has a history of growth through acquisition in virtually all of these areas. One of its largest acquisitions happened in March 2019 when Centene acquired WellCare for $17 billion. Since the acquisition, Centene merged Exactus Pharmacy Solutions, WellCare’s specialty pharmacy, into Acaria Specialty Pharmacy which they previously acquired in January 2013.

As of 2020, Centene ranked as the largest provider of government-sponsored health plans with roughly 22 million members. Centene ranked No. 42 in the 2020 Fortune 500 list of the largest United States corporations by total revenue.

Centene announced earlier this month that it also acquired a data analytics firm, Apixio, to support its pharmacy and health management operations, to expand Centene’s existing data analytics, and create the ability to document value-based payments. Value-based contracting, already a proof of concept, is thought to be a critical element to payer coverage of ultra-high cost specialty therapies (e.g., rare drugs) in coming years.

Centene and Specialty Pharmacy —–
Centene took its first deep dive into specialty pharmacy with the acquisition of Acaria Health, a specialty pharmacy in Orlando, FL. for $152million (a bargain price by today’s standards).

The announcement that Centene was doubling down on specialty pharmacy with the PantherRx deal should not be surprising given Centene’s robust acquisition history. PantherRx is a ripe acquisition. PantherRx was ranked as the 12th largest specialty pharmacy in 2019 with $1.2billion in sales. That is a big hunk of revenue and, in combination with the Acaria specialty pharmacy revenue, creates huge clout in the marketplace especially with manufacturers.

Manufacturers are key to fueling PantherRx’s revenue engine. PantherRx opened doors only in 2011 and grew rapidly with an aggressive marketing strategy pursuing new, limited-distribution drugs. These LD deals funneled thousands of new prescriptions to their doors.

PantherRx rebranded themselves in 2019 as PantherRx RARE to reinforce a niche in the exploding orphan drug / genetic therapy market. In 2020, virtually every FDA specialty drug approval has or will come to market through limited distribution. That’s called being in the right place at the right time.

Acaria has operated seamlessly in the marketplace under its original name since the Centene acquisition. Centene has announced that PantherRx Rare will also operate under current management and retain its name…. at least for the time being. Given the pull that the PantherRx name generates, Centene is smartly taking an “ain’t broke, don’t fix it approach”.

So, in closing, think about the bigger picture.

Over the past few years payers have taken the lead in owning/operating specialty pharmacies. We don’t know final numbers for 2020 but the top of the leaderboard includes the following payers: CVS/Aetna, CIGNA/ESI-Accredo, UHC/OptumRx, Humana, and now Centene. One might call that a trend, one that may soon reshape the PBM and specialty markets in unprecedented ways.

Centene Signs Definitive Agreement to Acquire PANTHERx Rare Pharmacy (PANTHERx)

ST. LOUIS, Dec. 15, 2020 /PRNewswire/ — Centene Corporation today announced it has signed a definitive agreement to acquire PANTHERx, one of the largest and fastest-growing specialty pharmacies in the United States specializing in orphan drugs and rare diseases. The transaction is subject to regulatory approvals and is expected to close by the end of 2020.

PANTHERx is a leader in rare disease pharmacy, comprehensively serving patients afflicted with rare and devastating conditions through delivery of medicine breakthroughs, clinical excellence, and access solutions. PANTHERx offers a suite of synchronized compliance, logistics, and analytics solutions to help streamline the process of delivering orphan medications and care to people living with complicated rare diseases.

“Centene has a long-standing commitment to providing care to the most underserved, complex populations,” said Michael F. Neidorff, Chairman, President and Chief Executive Officer for Centene. “PANTHERx adds a unique capability to our comprehensive pharmacy portfolio. We share a common goal of helping to remove barriers and reduce the burden for our members living with complex and rare diseases.”

“We are elated to enter a partnership that will propel us to the next level and provide opportunities for growth and stability while fostering an independence that delivers greater benefits to our patients, partners and associates,” stated Dr. Gordon J. Vanscoy, founder and CEO of PANTHERx. “Centene has committed to nurturing our patient-centric culture and focus on quality that is fundamental to PANTHERx’s tremendous success.”

PANTHERx and its management team will continue to operate independently as part of Centene’s Envolve Pharmacy Solutions, a total drug management program that includes integrated Pharmacy Benefit Manager (PBM) services and specialty pharmacy solutions to millions of members throughout the United States.


FDA Approves New IV Tx for Neuroblastoma – Danyelza

Someone must have lit a fire under the FDA new drug approval elves in the last few weeks as the activity level there suddenly boomed. There will be a lot of catch up to cover all the news in coming weeks.

In late November the FDA approved one of the very few new Biologics in 2020. The approval was for Danyelza (naxitamab) from Y-mAbs Therapeutics. The indication was for infusion in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for pediatric and adult patients with relapsed or refractory high-risk neuroblastoma.

Each year, about 800 children age 0 to 14 are diagnosed with neuroblastoma in the United States. It accounts for 6% of all childhood cancers in the United States. Almost 90% of neuroblastoma is found in children younger than 5.

Danyelza received an orphan drug designation as well as a priority review voucher for their rare pediatric disease product application. Pricing for Danyelza has still not yet been released. The MagellanRx Pipeline Report (October 2020) forecasts that the therapy will only generate $27 million in 2021 ramping up to $165 million in 2024….. small potatoes compared with some of the other 2020 approvals.

Given that Danyelza serves a very small patent population and a course of therapy will be costly, we expect that Danyelza will come to market as a distribution item, direct-to-infusion site, through a specialty pharmacy.

FDA grants accelerated approval to naxitamab for high-risk neuroblastoma in bone or bone marrow

On November 24, 2020, the Food and Drug Administration granted accelerated approval to naxitamab (DANYELZA, Y-mAbs Therapeutics, Inc.) in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for pediatric patients one year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy.

Efficacy was evaluated in patients with relapsed or refractory neuroblastoma in the bone or bone marrow enrolled in two single-arm, open-label trials: Study 201 (NCT 03363373) and Study 12-230 (NCT 01757626). Patients with progressive disease following their most recent therapy were excluded. Patients received 3 mg/kg naxitamab administered as an intravenous infusion on days 1, 3, and 5 of each 4-week cycle in combination with GM-CSF subcutaneously at 250 µg/m2/day on days -4 to 0 and at 500 µg/m2/day on days 1 to 5. At the investigator’s discretion, patients were permitted to receive pre-planned radiation to the primary disease site in Study 201 and radiation therapy to non-target bony lesions or soft tissue disease in Study 12-230.

The main efficacy outcome measures were confirmed overall response rate (ORR) per the revised International Neuroblastoma Response Criteria (INRC) and duration of response (DOR). Among 22 patients treated in the multicenter Study 201, the ORR was 45% (95% CI: 24%, 68%) and 30% of responders had a DOR greater or equal to 6 months. Among 38 patients treated in the single-center Study 12-230, the ORR was 34% (95% CI: 20%, 51%) with 23% of patients having a DOR greater or equal to 6 months. For both trials, responses were observed in either the bone, bone marrow or both.

The prescribing information contains a Boxed Warning stating that naxitamab can cause serious infusion-related reactions and neurotoxicity, including severe neuropathic pain, transverse myelitis and reversible posterior leukoencephalopathy syndrome (RPLS). To mitigate these risks, patients should receive premedication prior to each naxitamab infusion and be closely monitored during and for at least two hours following completion of each infusion.

The most common adverse reactions (incidence ≥25% in either trial) in patients receiving naxitamab were infusion-related reactions, pain, tachycardia, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection site reaction, edema, anxiety, localized edema, and irritability. The most common Grade 3 or 4 laboratory abnormalities (≥5% in either trial) were decreased lymphocytes, decreased neutrophils, decreased hemoglobin, decreased platelet count, decreased potassium, increased alanine aminotransferase, decreased glucose, decreased calcium, decreased albumin, decreased sodium and decreased phosphate.

The recommended naxitamab dose is 3 mg/kg/day (up to 150 mg/day) on days 1, 3, and 5 of each treatment cycle, administered after dilution as an intravenous infusion in combination with GM-CSF, subcutaneously at 250 µg/m2/day on days -4 to 0 and at 500 µg/m2/day on days 1 to 5. Treatment cycles are repeated every 4 to 8 weeks.

This application was granted accelerated approval based on overall response rate and duration of response. Continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials.

This application was granted priority review, breakthrough therapy, and orphan drug designation. A priority review voucher was issued for this rare pediatric disease product application. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.


Can a Limited Distribution Orphan Drug Not Be a Specialty Therapy?

We ran across a press release recently that looked a tad odd. It appeared that the FDA had approved a new therapy, Alkindi Sprinkle from Eton Pharma indicated as replacement therapy in pediatric patients with adrenocortical insufficiency (AI). But, this drug wasn’t appearing on any market reports for a pending new drug approval. Furthermore, the press release detailed that the drug was going into exclusive distribution via specialty pharmacy. Hmmmmmmm. A few minutes of internet sleuthing assuaged my confusion. It turns out that the approval was for a new formulation of a drug originally approved in 1952!!

But let’s look deeper into whether this drug qualifies as a specialty pharmacy item. We checked GoodRx and found that the cash price with coupon is as low as $215 for a month’s supply.
Whaaaaat????? That can’t be right. But it is.

So, does low price disqualify Alkindi Sprinkle as a specialty pharmacy product? Price has always been a wild card to determine if a drug is counted as a specialty product. But, throw in an Orphan Drug designation (AI affects between 5,000 and 11,000 children in the US) and the consensus has to lean towards awarding this drug a seat at the specialty pharmacy table. Don’t ya think?

This may help explain why Eton has placed it into limited distribution. The new formulation of Alkindi Sprinkle is a low dose oral granule option for children who, since 1952, were forced to rely on adult-strength hydrocortisone tablets to treat adrenocortical insufficiency. Low-dose options now allow for more accurate and individualized dosing for these kids. While there is no black box on the label, there are a few general warnings and precautions that might be of greater concern in children….. adding to the argument for going LD.

Alkindi Sprinkle is exclusively available through AnovoRx Specialty Pharmacy, yet another specialty pharmacy that is rebranding itself as a rare disease pharmacy.

Availability of Orphan Drug Alkindi Sprinkle (hydrocortisone) in the United States

DEER PARK, Ill., (GLOBE NEWSWIRE) — Eton Pharmaceuticals, Inc (Nasdaq: ETON), a specialty pharmaceutical company focused on developing and commercializing innovative treatments for rare pediatric diseases, today announced the full availability of Alkindi Sprinklefor sale and distribution in the United States. The U.S. Food and Drug Administration (FDA) has approved Alkindi Sprinkle (hydrocortisone) oral granules as replacement therapy for Adrenocortical Insufficiency (AI) in children under 17 years of age. Alkindi Sprinkle is the first and only FDA-approved granular hydrocortisone formulation for the treatment of adrenocortical insufficiency specifically designed for use in children.

Alkindi Sprinkle will be available exclusively through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. AnovoRx will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program will complete prescription fulfillment, insurance benefits investigation, educational support, aid qualified patients to obtain financial assistance along with other services designed to help patients access treatment. To enroll patients in the program and prescribe Alkindi Sprinkle, clinicians will need to complete a patient referral form available at

The FDA approval of Alkindi Sprinkle was supported by six clinical studies, including the first and only interventional Phase III study of oral hydrocortisone for Pediatric AI in neonates to children under eight years of age. Alkindi Sprinkle achieved significant increases in cortisol levels from baseline (P<0.0001) and was found to be well tolerated with no serious adverse events. Prior to the approval of Alkindi Sprinkle, oral hydrocortisone was only FDA-approved in tablet formulations of 5 mg and stronger. Many pediatric patients require significantly lower doses and the flexibility of precision titration. Alkindi Sprinkle will be available in 0.5-mg, 1-mg, 2-mg, and 5-mg strengths, allowing clinicians greater flexibility to individualize dosing based on each patient’s needs in accordance with the instructions for dosage and administration.

About Alkindi Sprinkle
Alkindi Sprinkle is an immediate-release oral hydrocortisone granule preparation that has been specifically designed to meet the dosing needs of pediatric patients with adrenocortical insufficiency. Prior to Alkindi Sprinkle’s approval, parent caregivers have had to cut or split higher-strength hydrocortisone tablets to achieve the lower doses required for small children, which could result in inaccurate dosing. Alkindi Sprinkle is manufactured using commercially proven technology in four strengths: 0.5 mg, 1 mg, 2 mg and 5 mg, to give greater dosing flexibility to clinicians. Taste-masking excipients that are acceptable for pediatric use eliminate the bitter taste of hydrocortisone. Alkindi Sprinkle has a shelf -life of three years at ambient temperature and does not require refrigeration.


FDA Approves First ORAL Tx for HAE – Orladeyo

Earlier this month the FDA approved yet another orphan drug, Orladeyo (berotralstat) from BioCryst Pharmaceuticals. It obtained an indication for the prevention of hereditary angioedema (HAE) attacks. Orladeyo is the first oral, non-biologic, once daily treatment for this condition.

HAE is caused by a genetic defect causing a biochemical imbalance that releases fluids outside of the blood vessels into surrounding tissues. Symptoms include swelling in various parts of the body, including the hands, feet, face and airway. Airway swelling can lead to death by asphyxiation. Before therapies became available, the mortality rate for airway obstruction was as high as 30%. HAE defect interferes with a blood protein (called C1 inhibitor) that helps to regulate blood-based systems involved in disease fighting, inflammation and coagulation.

Only 7,500 people are diagnosed and treated for HAE in the U.S.

Rare conditions are usually lucky to have a single novel therapy for patients. HAE is remarkably different as it has eight….. yep, eight brands to choose from. They include Berinert, Cinryze, Firazyr, Haegarda, Kalbitor, Orladeyo, Ruconest, Takhzyro. Oh, and a few days after the approval of Orladeyo the FDA approved a generic version of Firazyr. The more the merrier??

Orladeyo is a relatively late market entry and will compete with these well-established therapies. Leading competitive therapies include Cinryze and Haegarda, and Takhzyro. However, Cinryze requires intravenous (IV) administration, Haegarda is administered via subcutaneous injection every 3 to 4 days and Takhzyro is SC administered every 2 to 4 weeks. Orladeyo has the advantage of being the first oral formulation.

Specific pricing for Orladeyo was just announced. Many analysts believed that the non-biologic Orladeyo could be priced less than the biologic alternatives currently in use. But NO! Biocryst set the wholesale acquisition cost at $485,004 annually, or $37,308 per 28-day pack of either 150-mg or 110-mg capsules. By comparison, the WAC price for Takhzyro is ~$591,000 per year.

Biocryst announced that it has selected Optime Care Inc. as the exclusive ‘rare disease’ specialty pharmacy provider for Orladeyo. Patient shipments from Optime Care are expected to begin by the end of December.

BioCryst Announces FDA Approval of Orladeyo™ (berotralstat), First Oral, Once-daily Therapy to Prevent Attacks in Hereditary Angioedema Patients

RESEARCH TRIANGLE PARK, N.C., Dec. 03, 2020 (GLOBE NEWSWIRE) — BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that the U.S. Food and Drug Administration (FDA) has approved oral, once-daily Orladeyo™ (berotralstat) for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

“Orladeyo offers people with HAE and their physicians the first orally administered non-steroidal option for preventing HAE attacks and represents an important and welcome step in making more treatment options available to physicians and patients,” said Anthony J. Castaldo, president and chief executive officer of………



FDA Approves Sub-q Weight Loss Specialty Tx – Imcivree

Ok, we know you are ready for more catch up….. and it ain’t the red stuff.

The FDA recently approved yet another expensive specialty pharmacy therapy. The new arrival is called Imcivree (setmelanotide) and is from Rhythm Pharmaceuticals. It is indicated for chronic weight management caused by a bunch of genetic disorders that are too numerous to mention. The drug is designed to restore a biological pathway that, when disrupted, can lead to constant hunger.

Imcivree is provided as a subcutaneous injection. Although there are several unusual warnings and precautions included in the prescribing information, it was approved without a black box warning.

Non-prescription weight loss programs can be a bit pricey (I only hear rumors myself). Imcivree, however, breaks the bank by comparison. It will be priced at $330 per milligram for an adult at the recommended dose of 3 mg per day. That translates into an annualized cost of ~$360,000!

Given that the patient population is quite small, and the price is exceptionally high, Imcivree will certainly come to market through limited distribution.

FDA approves first treatment for weight management for people with certain rare genetic conditions

11/27/2020 — The U.S. Food and Drug Administration approved Imcivree (setmelanotide) for chronic weight management (weight loss and weight maintenance for at least one year) in patients six years and older with obesity due to three rare genetic conditions: pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, and leptin receptor (LEPR) deficiency confirmed by genetic testing demonstrating variants in POMC, PCSK1, or LEPR genes considered pathogenic (causing disease), likely pathogenic, or of uncertain significance. Imcivree is the first FDA-approved treatment for these genetic conditions.

Imcivree is not approved for obesity due to suspected POMC, PCSK1, or LEPR deficiency with variants classified as benign (not causing disease) or likely benign or other types of obesity, including obesity associated with other genetic syndromes and general (polygenic) obesity.

People with obesity due to POMC, PCSK1 or LEPR deficiency become severely obese at a young age. Patients are usually a normal weight at birth, but they gain excess weight because of a genetic defect (an imperfection or anomaly) that affects their level of hunger, ability to feel full or satiated, and energy output (metabolism). These conditions are very rare, with only around 150 reported in the medical literature for all three combined.

Imcivree works by activating areas in the brain that regulate appetite and fullness, causing patients with specific defects in these areas of the brain not to eat as much and to lose weight. The drug also increases resting metabolism (the number of calories the body burns at rest), which can contribute to weight loss. While Imcivree leads to weight loss in patients with obesity associated with these conditions, it does not treat the genetic defects that cause the conditions or other symptoms or signs.

Imcivree was evaluated in two 1-year studies. The first study enrolled patients with obesity and confirmed or suspected POMC or PCSK1 deficiency while the second study enrolled patients with obesity and confirmed or suspected LEPR deficiency; all patients were six years or older. The effectiveness of Imcivree was determined by the number of patients who lost more than 10 percent of their body weight after a year of treatment.

The effectiveness of Imcivree was assessed in 21 patients, 10 in the first study and 11 in the second. In the first study, 80 percent of patients with POMC or PCSK1 deficiency lost 10 percent or more of their body weight. In the second study, 46 percent of patients with LEPR deficiency lost 10 percent or more of their body weight.

The study also assessed the maximal (greatest) hunger in 16 patients over the previous 24 hours using an 11-point scale in patients 12 years and older. In both studies, some, but not all, of patients’ weekly average maximal hunger scores decreased substantially from their scores at the beginning of the study. The degree of change was highly variable among patients.

The most common side effects of Imcivree include injection site reactions, skin hyperpigmentation (skin patches that are darker than surrounding skin), headache and gastrointestinal side effects (such as nausea, diarrhea, and abdominal pain), among others. Spontaneous penile erections in males and adverse sexual reactions in females have occurred with treatment. Depression and suicidal ideation have also occurred with Imcivree.

FDA granted orphan disease designation, Breakthrough Therapy designation, and Priority Review for this drug application. FDA granted the approval of Imcivree to Rhythm Pharmaceutical, Inc.


FDA Approves Tx for Rare Genetic Disorder PH1 – Oxlumo

Ok, catch up time.
Looking back a couple of weeks….. the FDA approved yet another rare/orphan therapy (which is mostly all they they’ve approved in 2020!). The new approval is Oxlumo (lumasiran), from Swiss manufacturer Alnylam, as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder.

Patients with PH1 produce oxalate, a substance in food and produced by the body. It combines with calcium to cause kidney stones and deposits in the kidney, which can lead to kidney failure and dialysis. As the kidney function worsens, other organs including the heart, bones and eyes can be severely damaged. The disease is also difficult to diagnose, taking around six years before correctly identify it.

Oxlumo works by targeting hydroxyacid oxidase 1 mRNA that codes for the enzyme glycolate oxidase. Preventing the body from producing this enzyme has the effect of reducing the synthesis of oxalate.

Oxlumo is administered subcutaneous injection and is weight based dosed.

Like Alnylam’s other two marketed drugs, Oxlumo is expensive. The average annual list price is $493,000. The company expects that rebates and discounts will bring the average, effective price closer to only $380,000.

Surprise…. Surprise…. Oxlumo will come to market in open distribution…. said nobody ever.
As with so many 2020 approvals, Oxlumo will launch via limited distribution. Two specialty pharmacies working the rare specialty pharmacy segment have been confirmed….. Orsini Specialty Pharmacy and PantherRx Rare Pharmacy.


  • Disease: A specific/distinctive process in the body with specific cause and characteristic symptoms.
  • Disorder: Irregularity, disturbance, or interruption of normal functions. Medical disorders can be categorized into mental disorders, physical disorders, genetic disorders, emotional and behavioral disorders, and functional disorders.

FDA Approves First Drug to Treat Rare Metabolic Disorder

November 23, 2020 — The U.S. Food and Drug Administration has approved Oxlumo (lumasiran) as the first treatment for primary hyperoxaluria type 1 (PH1), a rare genetic disorder. This approval is a cumulation of the work of experts and community members coordinated by the Oxalosis & Hyperoxaluria Foundation and the Kidney Health Initiative.

“The approval of Oxlumo represents a great triumph of community involvement to address a rare disease. It is a result of input from patients, treating physicians, experts and sponsors at a patient-focused drug development meeting and through other collaborative efforts,” said Norman Stockbridge, M.D., Ph.D., director of the Division of Cardiology and Nephrology in the FDA’s Center for Drug Evaluation and Research.

Primary hyperoxalurias (PHs) are caused by excess production of oxalate, a substance consumed in food and also produced by the body. PH1 is the most common and severe type. PH1 affects an estimated one to three individuals per million in North America and Europe and accounts for approximately 80% of PH cases.

Patients with PH1 produce far too much oxalate, which can combine with calcium to cause kidney stones and deposits in the kidneys. Patients can experience progressive kidney damage, which can lead to kidney failure and the need for dialysis (a treatment that purifies the blood). As kidney function worsens, oxalate can build up and damage other organs, including the heart, bones and eyes.

Oxlumo works to decrease oxalate production. It was evaluated in two studies in patients with PH1: a randomized, placebo-controlled trial in patients six years and older and an open-label study in patients younger than six years. Patients ranged in age from four months to 61 years at the first dose. In the first study, 26 patients received a monthly injection of Oxlumo followed by a maintenance dose every three months; 13 patients received placebo injections. The primary endpoint was the amount of oxalate measured in the urine over 24 hours. In the Oxlumo group, patients had, on average, a 65% reduction of oxalate in the urine, compared to an average 12% reduction in the placebo group. By the sixth month of the study, 52% of patients treated with Oxlumo reached a normal 24-hour urinary oxalate level; no patients treated with the placebo did.

In the second study, 16 patients younger than six years all received Oxlumo. Using another measure of oxalate in the urine, the study showed, on average, a 71% decrease in urinary oxalate by the sixth month of the study.

The most common side effects of Oxlumo include injection site reaction and abdominal pain.

Oxlumo received orphan drug designation, which provides incentives to assist and encourage drug development for rare diseases. The application was also granted breakthrough therapy designation. In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children.

The FDA granted the approval of Oxlumo to Alnylam Pharmaceuticals, Inc.


Are Patients Getting Their Telehealth ‘Fix’

With the emergence of CV-19 just about every health care provider has been scrambling to do something to preserve their patient relationships. A patient that hangs alone on the vine with no tender loving care is one that may not only experience a decline in health but also a possible change in provider of care….. be it a hospital, a physician, or ‘oh no’ their pharmacy.

Specialty pharmacies have significantly ramped up patient outreach in recent years recognizing that more contact directly correlates with better outcomes, avoided adverse events, and better compliance and persistence. Telehealth is one of the methods that showed great promise as it is applicable to just about any disease / condition under the specialty pharmacy umbrella.

The article below goes further and suggests that telehealth goes together with rare and orphan diseases like a hand in glove. As stated, “A recent analysis found that telehealth visits increased 50% in March 2020, and a recent poll showed that more than 1 in 8 Americans engaged in a video consult with a health care professional in recent months. Those are strong numbers.

However, rare and orphan diseases are a lot more complicated and may not be so easy to support using the telehealth medium. For one, it demands that the health professional be well versed in the disease and all associated medications. That is a tall order even for most pharmacists and nurses. Non-clinicians simply can’t rely on even a well-crafted computer-based call scripts to ensure that the telehealth ‘visit’ passes clinical muster.

So, is there a role for telehealth in rare and orphan diseases clinical management? Yes.
We suggest that specialty pharmacies that work this segment well document their telehealth protocols and even measure the value from the effort….. and the expense. It will also go a long way to put a shine on their rare disease apple with patients, physicians, payers and, especially, manufacturers.

Pharmacy-Integrated Telehealth Solutions Support Patients with Orphan and Ultraorphan Disorders During and After COVID-19 Pandemic

2020-11-24 — During the coronavirus disease 2019 (COVID-19) pandemic, patients with orphan and ultra-orphan diseases have been challenged to get much-needed therapy adherence support. This means that 30 million Americans with a range of 7000 rare or orphan diseases—including Huntington disease, amyotrophic lateral sclerosis, Cushing syndrome, α1-antitrypsin, chronic immune thrombocytopenic purpura, and muscular dystrophy—have been at greater risk while self-protecting.

Many of these patients have struggled with reduced care, isolation, and adherence issues for most of their lives, but they now face elevated stressors because they are immunosuppressed, conscious of infection, and hypersensitive to viral threats. Adding to the challenge is that some pharmacies have reduced their hours. This impacts communication with physicians, which has already been severely disrupted due to the need for social distancing.

As an integral part of the care team, pharmacies serve as lifelines for these patients by helping to prevent lapses in continuity of care. For this reason, pharmacists who integrate with a telehealth solution can ensure that patients in rural locations—and those now living in self-imposed isolation—have consistent and reliable contact with pharmacy services during these unprecedented times.

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FDA Approves Oral Tx for Rare Genetic Condition in Kids – Zokinvy

The FDA recently revved up its approval engine waving yet another specialty pharmacy checkered flag for Zokinvy (lonafarnib) from Eiger BioPharmaceuticals. Zokinvy is indicated for a rare genetic condition, Hutchinson-Gilford progeria syndrome, and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. It is an oral formulation. Prior to this approval only supportive therapies were available for affected patients.

Progeria is caused by a point mutation in a single gene called lamin A. This mutated gene produces an abnormal form of the lamin A protein called progerin.

Progeria and Progeroid Laminopathies are serious, ultra-rare diseases characterized by premature and accelerated aging in children. Globally, there are an estimated 400 children with Progeria and another 400 children with Progeroid Laminopathies (in total, probably fewer than 200 in the US). Untreated, most patients will die from heart failure, heart attack or stroke. There is no known cure and the average life expectancy for the untreated children is around 14 years.

Zokinvy is dosed by height and weight, with an average dose of 175 milligrams per day; a bottle of 50-milligram and 75-milligram capsules will cost $21,500 and $32,000, respectively. That translates into an annual cost of $252,000 – $384,000.

Given the very small patient population and very high cost, it is almost assured that this therapy will be launched through exclusive distribution.

FDA Approves First Treatment for Hutchinson-Gilford Progeria Syndrome and Some Progeroid Laminopathies

SILVER SPRING, Md., Nov. 20, 2020 /PRNewswire/ — Today, the U.S. Food and Drug Administration approved Zokinvy (lonafarnib) capsules to reduce the risk of death due to Hutchinson-Gilford progeria syndrome and for the treatment of certain processing-deficient progeroid laminopathies in patients one year of age and older. Zokinvy is not approved for use in patients with other progeroid syndromes or laminopathies.

“Hutchinson-Gilford progeria syndrome and progeroid laminopathies are rare genetic diseases that cause premature aging and death and have a debilitating effect on people’s lives,” said Hylton V. Joffe, M.D., M.M.Sc, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research. “With today’s approval, Zokinvy is the first FDA-approved medication for these devastating diseases. The FDA will continue to work with stakeholders to advance the development of additional new, effective and safe therapies for these patients.”

Patients with Hutchinson-Gilford progeria syndrome and progeroid laminopathies experience accelerated cardiovascular disease from the buildup of defective progerin or progerin-like protein in cells. Before today’s approval, the only treatment options included supportive care and therapies directed towards the complications arising from the disease.

Zokinvy, a farnesyltransferase inhibitor, is an oral medication that helps prevent the buildup of defective progerin or progerin-like protein. The effectiveness of Zokinvy for the treatment of Hutchinson-Gilford progeria syndrome was demonstrated in 62 patients from two single-arm trials that were compared to matched, untreated patients from a separate natural history study. Compared to untreated patients, the lifespan of Hutchinson-Gilford progeria syndrome patients treated with Zokinvy increased by an average of three months through the first three years of treatment and by an average of 2.5 years through the maximum follow-up time of11 years. Zokinvy’s approval for the treatment of certain processing-deficient progeroid laminopathies that are very rare took into account similarities in the underlying genetic mechanism of disease and other available data.

The most common side effects included nausea vomiting, diarrhea, infection, decreased appetite and fatigue.

Zokinvy is contraindicated for co-administration with strong or moderate CYP3A inhibitors and inducers, as well as midazolam and certain cholesterol-lowering medications. Some patients treated with Zokinvy developed laboratory test abnormalities, such as changes in blood sodium and potassium levels, lowered white blood cell counts and increased liver blood tests. Routine blood laboratory testing should be obtained periodically. Eye toxicity was seen in animals so eye exams are recommended periodically and if there are new visual changes.

The FDA granted this application Priority Review designation. Zokinvy received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases, and Breakthrough Therapy Designation. In addition, the manufacturer received a rare pediatric disease priority review voucher. The FDA’s rare pediatric disease priority review voucher program is intended to encourage development of new drugs and biologics to prevent and treat rare diseases in children. The FDA granted the approval of Zokinvy to Eiger BioPharmaceuticals, Inc.

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