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Last week the FDA approved yet another biosimilar…… oh wait…. it was a biosimilar with an indication for not one, but TWO rare diseases. That makes yet another announcement of a new biosimilar a lot more interesting. 

The FDA approved Bkemv (eculizumab-aeeb), from Amgen, as the first interchangeable biosimilar to Soliris (eculizumab) to treat certain rare diseases with the same indicates as the reference product—–

• the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis; and 
• the treatment of patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy.

Bkemv is an infused therapy.

Bkemv also carries a Black Box warning for serious meningococcal infections.

PNH is an ultra-rare blood disorder that is characterized by the destruction of red blood cells. PNH occurs when the complement system — a part of the body’s immune system — over-responds, leading the body to attack its own red blood cells. Atypical hemolytic uremic syndrome is a rare genetic disease in which tiny blood clots form in blood vessels and block blood flow. It can lead to kidney failure and heart disease.

Bkemv is a monoclonal antibody that binds to the complement C5 protein and inhibits activation of the complement system, a part of the body’s immune system. This binding prevents the breakdown of red blood cells in the bloodstream (intravascular hemolysis) in patients with PNH and aHUS.

Under a 2020 settlement between the companies, Amgen will be free to launch its version March 1, 2025.

Amgen is not expected to release the price of Bekmv till launch time in 2025. By comparison, Soliris generally cost about $678,000 per year for the average patient. It will be interesting to see the competitive price.

Amgen did not disclose plans for distribution at this time.

CLICK HERE to access prescribing information

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FDA Approves First Interchangeable Biosimilar for Two Rare Diseases

May 28, 2024

CLICK HERE to access the press release

…………catching up on FDA approvals

The FDA recently approved a new oral solution, Livmarli (maralixibat) from Mirum Pharmaceuticals, Inc, indicated for the treatment of cholestatic pruritus in patients 3 months of age and older with Alagille syndrome (ALGS) and also for the treatment of cholestatic pruritus in patients 5 years of age and older with progressive familial intrahepatic cholestasis (PFIC).  Livmarli is an ileal bile acid transporter (IBAT) inhibitor. It was granted Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC.

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease typically leading to liver failure. In people with PFIC, liver cells are less able to secrete bile. The resulting buildup of bile causes liver disease in those affected. Signs and symptoms of PFIC typically include severe itching, jaundice, failure to thrive, and liver failure. The disease is estimated to affect fewer than 4,000 individuals in the US.

Published pricing for Livmarli oral solution (9.5 mg/mL) is about $56,240 for a supply of 30 milliliters.

Mirum did not announce plans for distribution. Given the small patient base, high cost and oral weight based dosing it is likely to be available through exclusive distribution.

CLICK HERE to access prescribing information

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Mirum Pharmaceuticals’ Livmarli Receives FDA Approval for Treatment of Cholestatic Pruritus in Patients with Progressive Familial Intrahepatic Cholestasis

CLICK HERE to access the press release

Our last few Reports focused on approvals and developments in the Cell and Gene Therapy (CGT) categories. A recent press release from a major national pharmacy chain would seem to reinforce the notion that CGT is the newest best thing since sliced bread.

Walgreens is going through a major remake that the company says is reflective of where the market is and, more importantly, where it is going. They thought it was going towards walk-in medical care which is now in ‘rethink’ mode which is a Report for another day.   But the strategists at WAGS now see CGT as a better bet….. one that is more aligned with their legacy pharmacy model and where the rare/orphan/ultra-orphan market appears to be heating up fast….. and where you need to be a serious player to maintain its ‘creds’ in the marketplace.

According to the press release, “Effective August 1, 2024, AllianceRx Walgreens Pharmacy will become Walgreens Specialty Pharmacy.” While this is a clean-up on its fractionated branding, WAGS is also planning on investing in CGT in a tangible way by opening a “Gene and Cell Services Pharmacy and Innovation Center – a dedicated 18,000-square-foot center in Pittsburgh, PA, with services and capabilities for these emerging therapies, including innovative solutions for managing the complexity of the supply chain, logistics and financing as well as clinical and social needs management to ensure success for patients and partners.” 

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Walgreens Launches Gene and Cell Services as Part of Newly Integrated Walgreens Specialty Pharmacy Business

Walgreens is expanding its specialty pharmacy services and investing in its capabilities as the company further grows its core pharmacy business to improve patient outcomes and provide greater value to payers and partners.

CLICK HERE to read the full press release

Today we are pleased to offer a review of an article from which just about anyone working in the specialty pharmacy segment might learn something…. or even a lot.

The topic is cell and gene therapy (CGT)….. which is getting increasingly greater attention given recent approvals and a jaw dropping pipeline.

Can you define cell therapy vs. gene therapy? 

Even now, many would struggle to clearly differentiate between them.

Here’s what the article says……

• Gene therapy is defined as the repair, replacement, or deactivation of dysfunctional genes to reestablish normal function. 
• Cell therapy is defined as the transfer of autologous (from oneself) or allogeneic (donor) cellular material into a patient for medical purposes.
• To devlop a CGT product a target gene or specific type of cell is identified to determine whether that gene or cell can be modulated directly within a patient’s own body (known as in vivo treatment), or if genetic or cellular material must be extracted from the body, modified, grown externally, and then transferred back into the patient (known as ex vivo treatment).
• Over the past decade+ the FDA has approved more than 30 cell, cord blood, and gene therapy products (see list in the article).
• Most investigational CGT products will continue to target oncologic conditions and rare diseases.
• More than 2,000 CGT products globally are in various stages of clinical development. and…. as we have all surely noticed by now,
• CGT therapies are among the most expensive medications in the world…….5 of the 10 most expensive drugs of 2023 were gene or cell therapies….. keeping health insurance companies awake at night worrying how to pay for them.

A reading of the full article will fill in much more detail of the current and, most importantly, give insight into the CGT ‘cures’ that one day are likely to reshape the legacy SP market. 

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Cell and Gene Therapies: A Review of Current and Future Treatments

CLICK HERE to read the full article

The rate of FDA approvals of gene therapies is picking up….. at a pace not too dissimilar to the bevy of biosimilars we’ve witnessed in recent years. The newest gene therapy is Lenmeldy (atidarsagene autotemcel) from Orchard Therapeutics. Lenmeldy is the first FDA-approved gene therapy that is indicated for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD). 

MLD is caused by a mutation on the ARSA gene, resulting in a deficiency of the enzyme arylsulfatase A (ARSA). This deficiency causes sulfatides to build up in cells and damage the central and peripheral nervous systems. As a result, patients experience a loss of motor and cognitive function and early death. MLD, which has no cure, is estimated to affect one in 40,000 individuals in the United States.

Lenmeldy is a one-time, individualized single-dose infusion made from the patient’s own hematopoietic (blood) stem cells (HSCs), which have been genetically modified to include functional copies of the ARSA gene. The stem cells are collected from the patient and modified by adding a functional copy of the ARSA gene. The modified stem cells are transplanted back into the patient where they engraft (attach and multiply) within the bone marrow.   The appropriate dose is calculated based on the child’s weight at time of Lenmeldy infusion using the instructions provided by the manufacturer. The processed dose is shipped to the treatment center in liquid nitrogen at less than -130°C (-202°F).

Lenmeldy now holds the austere position of being the most expensive therapy sold in the US. Orchard set the wholesale acquisition cost for Lenmeldy at $4.25 million.

CLICK HERE for prescribing information

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Gene Therapy for MLD Gets FDA Approval

CLICK HERE to read the full article

……. continuing to catch up on FDA approvals

The FDA recently approved a biosimilar, Simlandi (adalimumab-ryvk) from Alvotech and Teva Pharmaceuticals, as a biosimilar to Humira (adalimumab). Since there are already nine….. yep count ‘em….. other adalimumab biosims already approved, is there anything noteworthy to say about Simlandi…. YES!

The noteworthy thing about this approval is that Simlandi was approved with the coveted interchangeable designation. So, the ability for pharmacist substitution without physician approval in many states gives Simlandi a tangible marketing edge. However, the FDA had already approved Pfizer’s Abrilada (adalimumab-afzb) and Boehringer Ingelheim’s Cyltezo (adalimumab-adbm). The more the merrier?

But wait!…. Simlandi is high-concentration as well as citrate-free giving it a slight marketing edge over its interchangeable competitors….. as well as the rest of the pack.

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FDA Approves Simlandi, Third Interchangeable Humira Biosimilar

CLICK HERE to read the full press release

There were no big surprises in the recently published list of the Top 15 specialty pharmacies nationally. That is if you only look at the ranking order and are not looking at revenues….. which continue to grow and grow generating about $242 billion in overall sales in 2023.

Here is the recently published list…….

Top 15 specialty pharmacies by 2023 revenue

Key takeaways—–

• Two in five accredited specialty pharmacies are owned by hospitals and other healthcare providers
• The nation’s three biggest pharmacy benefit managers accounted for 67% of specialty pharmacy revenue in 2023. 
• Hospitals and health systems are the fastest-growing participants in this market, partly because of drugmakers’ restrictions regarding the 340B program, the report said. 
• This year’s list included “Other retail, mail, long-term care and specialty pharmacies” as a group that  garnered over $37 billion of the total ~$242 billion or about 15%.

1. CVS Specialty (CVS Health) — $73.3 billion

2. Accredo/Freedom Fertility (Cigna/Evernorth/Express Scripts) — $59.5 billion

3. Other retail, mail, long-term care and specialty pharmacies — $37.4 billion

4. Optum Specialty Pharmacy (UnitedHealthGroup/OptumRx) — $32.3 billion

5. AllianceRx Walgreens Pharmacy/Walgreens stores — $8.4 billion

6. CenterWell Specialty Pharmacy (Humana) — $6.2 billion

7. Onco360 Pharmacy/CareMed Specialty Pharmacy (BrightSpring Health Services) — $4.6 billion

8. PANTHERx Specialty Pharmacy — $3.6 billion

9. Walmart Specialty Pharmacy/Walmart stores — $3.4 billion

10. Kroger Specialty Pharmacy/Kroger stores — $3.2 billion

11. Acaria Health (Centene) — $2.9 billion

12. Biologics/RxCrossroads (McKesson) — $2.2 billion

13. SenderraRx — $2 billion

14. AHF Pharmacy (AIDS Healthcare Foundation) — $1.9 billion

15. Lumicera Health Services (Navitus Health Solutions) — $1.5 billion

CLICK HERE to access the full article

The FDA recently approved a ‘two-fer’…. yep, TWO identical biosimilars with the approval of Jubbonti (denosumab-bbdz) and Wyost (denosumab-bbdz) from Sandoz, Inc. They are the same formulations, and both received the coveted interchangeable designation. They are the first interchangeable biosimilars for RANKL inhibitors. Both therapies are available for subcutaneous administration. 

Jubbonti is indicated for the following (which are also currently approved for Prolia.)

• postmenopausal women with osteoporosis at high risk for fracture;
• increasing bone mass in men with osteoporosis at high risk for fracture;
• glucocorticoid-induced osteoporosis in men and women at high risk for fracture;
• increasing bone mass in men at high risk for fracture receiving androgen deprivation therapy for nonmetastatic prostate cancer; and
• increasing bone mass in women at high risk for fracture receiving adjuvant aromatase inhibitor therapy for breast cancer.

Wyost is indicated for the following (which are also currently approved for Xgeva.)

• prevention of skeletal-related events in patients with multiple myeloma and in patients with bone metastases from solid tumors;
• treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity; and
• treatment of hypercalcemia of malignancy refractory to bisphosphonate therapy.

CLICK HERE to access prescribing information for both therapies.

Sandoz has yet to confirm pricing. 

Similarly, distribution details for either/both therapies have not been disclosed.

Announcements for newly approved specialty drugs often state that the product will be available through specialty pharmacy in limited distribution. However, the early press releases rarely specify the specialty pharmacy(ies) selected as the designated partner(s).

Here are four LD deals that have been publicly confirmed subsequent to their approvals.

ONCO360 to distribute Voydea

Onco360 has been selected as a national pharmacy partner by Alexion Pharmaceuticals for Voydeya (danicopan) as a first class oral, Factor D inhibitor for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) in combination with C5 inhibitor, Ultomiris (ravulizumab) or Soliris (eculizumab) for patients that experience clinically significant extravascular hemolysis (EVH).1 Voydeya® is an oral, Factor D inhibitor that acts in the alternative complement pathway of the immune system, providing control of red blood cell destruction within and outside the blood vessels.

ONCO360 to distribute Ojemda

Onco360 has been selected as a pharmacy partner by Day One Biopharmaceuticals for Ojemda (tovorafenib), which is now FDA approved for the treatment of patients six months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.

Orsini Specialty Pharmacy to Dispense Yargesa

Orsini Specialty Pharmacy has been selected by Edenbridge Pharmaceuticals as the preferred specialty pharmacy partner for the distribution of Yargesa (miglustat) capsules, 100mg. YARGESA is a glucosylceramide synthase inhibitor indicated for the treatment of adult patients with Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.

PANTHERx Rare to distribute Yargesa

PANTHERx Rare announced that it was selected by Edenbridge Pharmaceuticals for the distribution of Yargesa (miglustat) capsules, the first oral treatment option for adults with mild to moderate Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option.

The FDA recently approved yet another – first in class -cellular therapy, Amtagvi (lifileucel) from Iovance Biotherapeutics Inc., indicated for the treatment of adult patients with a type of skin cancer (melanoma) that is unable to be removed with surgery (unresectable) or has spread to other parts of the body (metastatic) that previously has been treated with other therapies.

Exposure to ultraviolet light is the leading cause of Melanoma, a form of skin.  Melanomas represent approximately 1% of all skin cancers yet are related to a significant number of cancer-related deaths as they metastasize. 

Amtagvi is a tumor-derived autologous T cell immunotherapy composed of a patient’s own T cells. Tumor tissue from the patient is surgically removed and their T cells are separated from the sample. The resulting therapy is then infused in the patient as a single dose. This is the first FDA-approved tumor-derived T cell immunotherapy.

Amtagvi was approved with a Boxed Warning due to prolonged severe low blood count, severe infection, cardiac disorder, respiratory or renal function failure. 

CLICK HERE for prescribing information

The company confirmed that Amtagvi will carry a list price of $515,000.

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FDA Approves First Cellular Therapy to Treat Patients with Unresectable or Metastatic Melanoma

CLICK HERE to read the full article