Month: October 2023

Catching up on recent FDA approvals.

The FDA recently approved a new oral therapy, Sohonos (palovarotene) from Ipsen Biopharmaceuticals, for reduction in the volume of new heterotopic ossification (extra-skeletal bone formation) in adults and children aged 8 years and older for females, and 10 years and older for males with fibrodysplasia ossificans progressiva.  Sohonos is the first drug approved for patients with this condition.

Fibrodysplasia ossificans progressiva is a rare, autosomal dominant disease where connective tissue such as muscle, tendons and ligaments gradually turn into bone tissue, causing limited movement, deformities and severe disability. Sohonos is a retinoic acid receptor gamma agonist that curbs progression of the genetic disease by reducing the volume of new, abnormal bone growth.

US approval followed four years in application queue, two clinical trial holds, a complete response letter, a European Commission rejection, a scrapped trial in another indication and multiple safety flags, resubmissions, and other delays.

Ipsen confirmed that Sohonos will launch at a cost of $624,000 annually for a 5-mg daily dose for an ‘average’ patient….. but dosing will also depend on disease severity and patient weight.

Ipsen did not disclose distribution plans for the launch. Given its ‘rare’ designation, cost, and management challenges, it is expected that distribution will be through limited distribution.

Catching up on recent approvals……..

The FDA recently approved a new oral therapy, Akeega (niraparib and abiraterone acetate) from Janssen Pharmaceutical, the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive metastatic castration-resistant prostate cancer (mCRPC), as detected by an FDA-approved test.

As noted in the press release prostate cancer is one of the most common cancers in the U.S., with an estimated 288,300 new cases and nearly 35,000 deaths expected in 2023. Upwards of 15 percent of patients with mCRPC have BRCA gene alterations. Patients with BRCA-positive mCRPC are more likely to have aggressive disease and may experience poor outcomes and a shorter survival time.

The recommended starting dose is 200 mg niraparib/1,000 mg abiraterone acetate (two tablets). The 100 mg niraparib/1,000 mg abiraterone acetate dose option (two tablets) is available for dose reduction.

Janssen did not confirm the cost of Akeega at the time of approval, however, its cost is listed at $9,600 with coupon through GoodRx.

Janssen did not announce distribution details at the time of approval but at least one specialty pharmacy, Onco360, has since confirmed that they are at least one provider selected to be a limited distribution partner for Akeega.

CLICK HERE for full prescribing details

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U.S. FDA Approves Akeega (Niraparib and Abiraterone Acetate), the First-And-Only Dual Action Tablet for the Treatment of Patients with BRCA-Positive Metastatic Castration-Resistant Prostate Cancer

Approval is based on the Phase 3 MAGNITUDE study, a prospectively designed precision medicine study including the largest population of BRCA-positive patients in combination trials to date with metastatic castration-resistant prostate cancer (mCRPC)

Akeega plus prednisone significantly improved radiographic progression-free survival compared to abiraterone acetate plus prednisone (AAP) in patients with BRCA-positive mCRPC

HORSHAM, Pa., – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the U.S. Food & Drug Administration (FDA) has approved Akeega (niraparib and abiraterone acetate), the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive mCRPC, as detected by an FDA-approved test.

CLICK HERE to read the full Janssen press release

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

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

Biologics to Dispense Vanflyta

Biologics by McKesson was selected by Daiichi Sankyo, Inc., as a specialty pharmacy provider for Vanflyta (quizartinib).  Vanflyta is indicated for use in combination with standard cytarabine and anthracycline induction and cytarabine consolidation, and as maintenance monotherapy following consolidation chemotherapy, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FLT3-ITD positive as detected by an FDA-approved test. 

Orsini Specialty Exclusive Distributor for Veopoz

Orsini Specialty Pharmacy announced that Regeneron Pharmaceuticals, Inc. has chosen Orsini to be the exclusive specialty pharmacy partner for Veopoz (pozelimab-bbfg). A monoclonal antibody, Veopoz is the first and only treatment for those living with CHAPLE disease, an ultra-rare and life-threatening hereditary disease.

Orsini Specialty to Dispense Pombiliti / Opfolda

Orsini Specialty Pharmacy has been selected by Amicus Therapeutics to dispense Pombiliti and Opfolda, a two-component treatment approved for certain patients with late-onset Pompe disease. Pombiliti is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency).

Nufactor Exclusive Specialty Pharmacy for Ycanth

Nufactor, Inc., a subsidiary of FFF Enterprises Inc., announced that it has partnered with Verrica Pharmaceuticals to be the exclusive specialty pharmacy to dispense Ycanth, the first FDA approved treatment of molluscum contagiosum in adult and pediatric patients 2 years of age and older.

Onco360 to Dispense Ojjaara

Onco360 has been selected as a pharmacy partner by GlaxoSmithKline for Ojjaara (momelotinib) a kinase inhibitor indicated for the treatment of intermediate or high-risk myelofibrosis (MF), including primary MF or secondary MF [post-polycythemia vera (PV) and post-essential thrombocythemia (ET)], in adults with anemia.

Onco360 to Dispense Akeega

Onco360 has been selected as a pharmacy partner by The Janssen Pharmaceutical Companies of Johnson & Johnson for Akeega (niraparib and abiraterone acetate). Akeega is indicated with prednisone for the treatment of adult patients with deleterious or suspected deleterious BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC).

CarePartners Pharmacy named exclusive distributor of Yuflyma

CarePartners Pharmacy has been named the exclusive distributor for Yuflyma, a biosimilar to Humira. Yuflyma was approved by the FDA in May, 2023. by Celltrion Healthcare, Celltrion’s distribution unit, said Monday it has signed a deal with US specialty pharmacy chain CarePartners Pharmacy to sell its Humira biosimilar Yuflyma in the US market. Celltrion inked an exclusive contract with CarePartners.

It’s ALIVE!!!  Artificial Intelligence is here.

The article below details how HCSC, which covers about 18 million members in Illinois, Montana, New Mexico, Oklahoma and Texas, has introduced the power of AI to fast-track their very hectic prior auth volume. The company received about 1.5 million prior authorization requests in 2022.

What is most impressive is that they claim prior auths can be processed 1,400 times faster than using the traditional method.  WOW! They say that AI technology is now used for 93% of HCSC members for a limited number of procedure codes while they ramp up AI to handle even more procedure codes. 

HCSC also developed  an algorithm that mines historical prior authorization approvals to make it easier to approve treatment within seconds. If the request for a prior auth meets all criteria it is approved with no human intervention. The requests that fall through the sifter then go into a traditional prior authorization review.

Will AI completely eliminate manual review?

— Unlikely, at least in the foreseeable future.

Specific procedure codes and requests for certain (many?) specialty medications are likely to still be subject to lots of review involving extra documentation such as lab tests, genetic testing, site of service, etc. etc. It is conceivable that even these elements could be automated….. but given the difficulty in linking all these disparate criteria into one platform will be a big challenge in the short term.

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How HCSC is using AI to speed up prior authorization

Health Care Service Corporation says using augmented intelligence allows it to process prior authorization requests about 1,400 times faster than before. 

Jul 17, 2023  – Of the many tools that payers use to control costs, prior authorization ranks high on the list of what providers and patients find particularly vexing. However, Health Care Service Corporation hopes to fine-tune the process using the power of artificial intelligence. —–article continues—–

CLICK HERE to access the complete article

Catching up on a slew of FDA approvals in recent weeks….. whew!

The FDA  recently approved a new therapy, Veopoz (pozelimab-bbfg) from Regeneron, for the treatment of adult and pediatric patients 1 year of age and older with Chaple disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for Chaple. Administration is by intravenous or subcutaneous injection.

Chaple is an ultra-rare and life-threatening hereditary immune disease driven by an overactivation of the complement system. In healthy individuals, the complement system is a mechanism for destroying microbes. However, those living with Chaple are unable to regulate complement activity due to mutations in their CD55 gene. Without proper CD55 regulation, the complement system may attack normal cells, causing damage to blood and lymph vessels along the upper digestive tract and leading to the loss of circulating proteins. 

There are fewer than 10 patients with Chaple disease diagnosed in the U.S.

Regeneron confirmed that the cost of Veopoz will be $34,600 per vial. 

Veopoz will launch through specialty distribution. 

CLICK HERE to access prescribing information

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VEOPOZ (POZELIMAB-BBFG) RECEIVES FDA APPROVAL AS THE FIRST TREATMENT FOR CHILDREN AND ADULTS WITH CHAPLE DISEASE

CHAPLE is an ultra-rare hereditary disease that can cause potentially life-threatening gastrointestinal and cardiovascular symptoms

Approval represents 10th FDA-approved medicine invented by Regeneron

With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg biologics license application (BLA) have been addressed

FDA action on the aflibercept 8 mg BLA is expected in the next few weeks

TARRYTOWN, N.Y., Aug. 18, 2023  — Regeneron Pharmaceuticals, Inc. announced the U.S. Food and Drug Administration (FDA) has approved Veopoz (pozelimab-bbfg) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease, also known as CD55-deficient protein-losing enteropathy. Veopoz is the first and only treatment indicated specifically for CHAPLE. With the approval of Veopoz, the pre-approval inspection issues related to the aflibercept 8 mg BLA have been addressed. FDA action on the aflibercept 8 mg BLA is expected in the next few weeks.

……….article continues…………..

CLICK HERE to access the full press release

Catching up on recent approvals…..

Earlier this week we sent a Report on the approval of Talvey from Janssen Pharmaceuticals indicated for he treatment of adult patients with relapsed or refractory multiple myeloma. Within days the FDA issued approval for another subcutaneous therapy, Elrexfio (elranatamab-bcmm) from Pfizer, Inc. Talvey was a first-in-class bispecific antibody.

Like Talvey, Elrexfio is a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, for adults with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.

Similarly, approval of Elrexfio included a Black Box Warning for cytokine release syndrome (CRS) and neurologic toxicity including immune effector cell-associated neurotoxicity syndrome (ICANS). The assigned REMS program includes monitoring requirement for oral toxicity, weight loss, infections, cytopenias, skin toxicity, hepatoxicity and embryo-fetal toxicity. Seeing a trend here?

Approval requires that patients be hospitalized for 48 hours after all doses within the step-up dosing schedule. Another coincidence to be sure.

Pfizer did not disclose details for distribution or logistics at the time of approval. 

The company did confirm that Elrexfio will launch at a cost of $41,500 per month (about $330,000 annually). This compares with Talvey’s launch price of $45,000 per month.

Given its cost, complex dosing schedule and multiple risks, Elrexfio will likely be handled through specialty pharmacy distribution.

CLICK HERE for full prescribing information

Catching up on recent approvals…..

The FDA recently approved a new subcutaneous therapy, Talvey (talquetamab-tgvs) from Janssen Pharmaceutical (a J&J company) for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. Talvey is a first-in-class bispecific antibody.

Approval included a Black Box Warning for cytokine release syndrome (CRS) and neurologic toxicity including immune effector cell-associated neurotoxicity syndrome (ICANS). The assigned REMS program includes monitoring requirement for oral toxicity, weight loss, infections, cytopenias, skin toxicity, hepatoxicity and embryo-fetal toxicity.

Approval requires that patients be hospitalized for 48 hours after all doses within the step-up dosing schedule.

Janssen did not disclose details for distribution or logistics at the time of approval. 

The company did confirm that Talvey will launch at a cost of $45,000 per month.

Given its cost, complex dosing schedule and multiple risks Talvey is expected to be channeled through specialty pharmacy distribution. 

CLICK HERE to access prescribing information

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U.S. FDA Approves Talvey (talquetamab-tgvs), a First-in-Class Bispecific Therapy for the Treatment of Patients with Heavily Pretreated Multiple Myeloma

Bispecific antibody targeting GPRC5D receptor showed an overall response rate of more than 70 percent with durable responses, including in patients previously treated with a bispecific antibody or CAR-T cell therapy

HORSHAM, Pa. /PRNewswire/ — The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of Talvey (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.1 This indication is approved under accelerated approval based on response rate and durability of response.1 Continued approval for this indication is contingent upon verification and description of clinical benefit in confirmatory trial(s).  article continues……….

CLICK HERE to read the full press release