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FDA Update for Week of April 22, 2024: Second Gene Therapy for Hemophilia B


This week, the FDA has approved several products, including Beqvez, the second gene therapy hemophilia B; Ojemda for children with brain tumors; an additional indication for Lutathera for adolescents with neuroendocrine tumors; and a bladder cancer immunotherapy. The agency has also issued a complete response letter for pz-cel to treat epidermolysis bullosa.

FDA Approves Pfizer’s Gene Therapy for Beqvez for Hemophilia B

The FDA has approved Pfizer’s one-time gene therapy Beqvez (fidanacogene elaparvovec-dzkt) to treat adults with moderate-to-severe hemophilia B. It is indicated for those who currently use factor IX (FIX) prophylaxis therapy, have current hemorrhage, repeated bleeding episode or do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid.

Hemophilia is a rare genetic bleeding disorder caused by a deficiency in one of several blood clotting factors. People with hemophilia B have a deficiency in clotting human coagulation Factor IX, a specific protein in the blood.

Beqvez is a one-time gene therapy that contains a bio-engineered adeno-associated virus (AAV) capsid and a high-activity variant of the Factor IX gene. Pfizer licensed the gene therapy from Spark Therapeutics. Bequez will be priced at $3.5 million, which is in line with the previously approved gene therapy for hemophilia B Hemgenix.

A Pfizer spokesperson said the price is based on a holistic view of its value, including the benefits generated for patients, healthcare systems, and society. Annual costs per individual may be more than $600,000 and as high as $1.1 million, not including indirect costs such as outpatient care and managing uncontrolled bleeds.

Pfizer executives said they are working with treatment centers, payers, and the hemophilia community to ensure access. Pfizer is launching a warranty program based on durability of patient response to treatment with the goal of offering financial protection by insuring against the risk of efficacy failure, the news release said.

The spokesperson said Pfizer will continue to provide coverage if a patient changes insurance after they receive treatment for commercially insured patients, and the company anticipates a refund will be provided to the insurers. “This was informed by pre-approval information exchange discussions we have had with payers to understand how we can best ensure access.

FDA Approves Ojemda for Children with Brain Tumors

The FDA has granted accelerated approval to Day One’s Ojemda (tovorafenib), to treat children 6 months of age and older with relapsed or refractory low-grade glioma. It is indicated for patients who have a BRAF fusion or rearrangement, or BRAF V600 mutation.

Pediatric low-grade glioma (pLGG) is the most common brain tumor diagnosed in children. BRAF is the most commonly altered gene in pLGG, with up to 75% of children having a BRAF alteration.

Ojemda is a type II RAF kinase inhibitor of mutant BRAF V600, wild-type BRAF, and wild-type CRAF kinases. It is available as a tablet or an oral suspension and is administered once weekly. Until now, there had been no medicines approved for patients with pediatric low-grade glioma with BRAF fusions.

During an investor call, Lauren Merendio, chief commercial officer of Day One, said they expect 60% of patients eligible for Ojemda will have commercial insurance. They expect the remainder of patients to be in Medicaid programs.

Ojemda will have a wholesale acquisition cost of $33,916 for a 28 day supply and will be distributed through specialty pharmacies Biologics by McKesson and Onco360.

FDA Approves Lutathera for Adolescents with Neuroendocrine Tumors

The FDA has approved Novartis’ Lutathera (lutetium Lu 177 dotatate) to treat adolescents 12 years of age and older with somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Lutathera is already approved to treat adults with these tumors.

Neuroendocrine tumors are a type of cancer that originates in neuroendocrine cells throughout the body and are commonly considered slow-growing malignancies. About 10% to 20% of pediatric patients are diagnosed with metastatic disease.

Lutathera is a radiotherapeutic that was approved in 2018 to treat adults with gastroenteropancreatic neuroendocrine tumors. It belongs to a class called Peptide Receptor Radionuclide Therapy (PRRT), a form of targeted treatment comprised of a targeting molecule that carries a radioactive component. The targeting molecule binds to a specific receptor on tumor cells, and is then internalized into the target cells, where the radioactive component destroys the tumor cells.

The list price (wholesale acquisition cost) of Lutathera is $55,896, and most patients require about four doses, according to a company spokesperson. The dosage is the same for adults and adolescents.

Novartis offers a $25 copay program for commercially insured patients up to a maximum annual benefit of $15,000. The terms state: “The value of this Program is exclusively for the benefit of patients and is intended to be credited toward patient out-of-pocket obligations and maximums, including applicable co-payments, coinsurance, and deductible.”

For the full year 2023, Lutathera generated sales of $605 million, up 28% from 2022.

FDA Approves Bladder Cancer Immunotherapy

The FDA has approved the biologics license application (BLA) for ImmunityBio’s Anktiva (N-803, or nogapendekin alfa inbakicept-pmln) in combination with Bacillus Calmette-Guérin (BCG) vaccine. Anktiva, an antibody cytokine fusion protein, is approved to treat BCG-unresponsive non-muscle invasive bladder cancer.

The American Cancer Society estimates there will be about 83,190 new cases of bladder cancer in 2024, with 16,840 deaths.

The cytokine interleukin-15 (IL-15) plays a crucial role in the immune system by affecting the development, maintenance, and function of the natural killer and T cells. Anktiva, an IL-15 superagonist, has a mechanism of action that leads to the proliferation of natural killer and T cells. This provides a boost to the immunological response generated by BCG.

Anktiva will be available mid-May 2024. No information was provided about pricing.

FDA Issues Complete Response Letter for Pz-Cel to Treat Epidermolysis Bullosa

The FDA has issued a complete response letter for the biologics license application (BLA) for prademagene zamikeracel (pz-cel) to treat patients with recessive dystrophic epidermolysis bullosa (RDEB). RDEB is rare connective tissue disorder. The disorder causes severe and painful skin wounds and can lead to systemic complications. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen.

Developed by Abeona Therapeutics, pz-cel is a cell therapy designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells. The FDA is asking for additional information for the Chemistry Manufacturing and Controls (CMC) requirements.

Company officials said in a news release that they had planned to provide CMC data prior to BLA approval, with plans to provide full validation reports after approval in mid-2024. FDA officials, however, said in the CRL that he proposed timing of the data submission would not allow sufficient review before the May 25, 2024, PDUFA date.

Abeona said the information requested by the FDA pertains to validation requirements for certain manufacturing and release testing methods. The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA. Regulators did not request any new clinical trials or clinical data to support the approval of pz-cel.

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