FDA Updates for Week of June 26, 2023

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Approvals this week include: a $2.9 million gene therapy for hemophilia A; a pediatric growth hormone; the first cell therapy for type 1 diabetes; a new alopecia drug; a therapy for generalized myasthenia gravis; and full approval for Blincyto in B-cell ALL. The agency also rejected a higher dose of Eylea and set an action for hemophilia B gene therapy.

FDA Approves $2.9 Million Gene Therapy for Hemophilia A

The FDA has approved BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) to treat adults with severe hemophilia A. The one-time, single-dose infusion is the first approved gene therapy for severe hemophilia A. Hemophilia A is a genetic disease caused by the deficiency of clotting factor VIII.

Roctavian is designed to replace the function of the mutated gene, allowing people with severe hemophilia A to produce their own Factor VIII and thereby limit bleeding episodes. The gene therapy uses an adeno-associated virus vector to deliver a functional gene to the cells in the liver, allowing the body to produce its own Factor VIII.

Roctavian, which will available in about eight weeks, will have a wholesale acquisition cost of $2.9 million based on the weight of a typical patient, the company said in an investor call. The company indicated that Roctavian adds value in terms of both the clinical benefit it offers patients and the economic benefit, which is about $800,000 a year for the typical patient.

BioMarin is offering an outcomes-based warranty that will reimburse government and commercial payers up to 100% of wholesale acquisition cost in the event that a person does not respond to Roctavian. If an individual treated with Rocktavian loses response at any time in the first four years after dosing, BioMarin will reimburse payers on a prorated basis for the cost of treatment.

New Pediatric Growth Hormone Therapy Costs $8,300 Monthly

Pfizer and OPKO Health’s new pediatric growth hormone treatment, Ngenla (somatrogon-ghla), approved by the FDA on June 28, has a monthly wholesale acquisition cost (WAC) of $8,300, a Pfizer spokesperson said.

Ngenia, which will be available in August, is a once-weekly, human growth hormone analog for pediatric patients 3 years and older who have growth failure due to inadequate secretion of endogenous growth hormone. The price is generally in line with other once weekly growth hormone treatments, the spokesperson said.

Ngenla offers a new treatment option for children living with growth hormone deficiency. “Currently the standard of care is a once-daily injection administered subcutaneously. It is estimated that up to two-thirds of children with GHD may miss more than one daily dose per week.

FDA Approves First Cellular Therapy for Type 1 Diabetes

The FDA has approved CellTrans’ Lantidra (donislecel), the first allogeneic (donor) pancreatic islet cellular therapy to treat patients with type 1 diabetes. It is approved for patients who unable to achieve target glycated hemoglobin.

Type 1 diabetes is a chronic autoimmune disease that requires daily administration of insulin. The primary mechanism of action of Lantidra is believed to be the secretion of insulin by the infused allogeneic islet beta cells. It is administered as a single infusion into the liver portal vein. An additional infusion of Lantidra may be performed depending on the patient’s response to the initial dose.

The safety and effectiveness of Lantidra was evaluated in two single-arm studies with 30 patients with type 1 diabetes. Overall, 21 participants did not need to take insulin for a year or more, with 11 participants not needing insulin for one to five years and 10 participants not needing insulin for more than five years. Five participants did not achieve any days of insulin independence.

FDA Approves Rystiggo for Generalized Myasthenia Gravis

The FDA has approved UCB’s Rystiggo (rozanolixizumab-noli) to treat adults with generalized myasthenia gravis (gMG). It is be indicated for patients who are positive for the anti-acetycholine receptor (AChR) or the anti-muscle-specific tyrosine kinase (MuSK) antibody.

Generalized myasthenia gravis is a chronic and unpredictable autoimmune disease that causes weakness in the skeletal muscles, including those in the arms and legs and those involved in breathing.

Rystiggo is a subcutaneous (under the skin) monoclonal antibody that targets the neonatal Fc receptor (FcRn). It has been designed to block the interaction of Fc receptor and Immunoglobulin G (IgG), accelerating the catabolism of antibodies and reducing the concentration of IgG autoantibodies. It will be commercially available in the third quarter of 2023 and will have a list price of $6,050 per vial. For patients without insurance, the company offers financial assistance.

FDA Converts Blincyto to Full Approval for Minimal Residual Disease in B-Cell ALL

The FDA has converted the accelerated approval of Blincyto (blinatumomab) to a full approval to treat adults and pediatric patients with CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL). It is indicated for patients in first or second remission with minimal residual disease (MRD). B-cell precursor acute lymphoblastic leukemia is a fast-growing cancer in which many B-cell lymphoblasts are found in the bone marrow and blood.

Developed by Amgen, Blincyto is a BiTE (bispecific T-cell engager) immuno-oncology therapy that targets CD19 surface antigens on B cells that was granted accelerated approval in 2018 for patients with minimal residual disease. Its current wholesale acquisition cost price is $4,900.15 per vial, according to an Amgen spokesperson.

BiTE molecules fight cancer by helping the body's immune system detect and target malignant cells by engaging T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. By bringing T cells near cancer cells, the T cells can inject toxins and trigger cancer cell death.

FDA Approves Pfizer’s Alopecia Drug

The FDA has approved Pfizer’s Litfulo (ritlecitinib), a once-daily oral treatment, for people 12 years of age and older with severe alopecia. Alopecia is an autoimmune disease characterized by patchy or complete hair loss on the scalp, face, or body. It develops when the immune system attacks the body’s hair follicles, causing hair to fall out. It impacts almost 7 million in the United States, and 20% of them are diagnosed before the age of 18.

Litfulo is a kinase inhibitor, which inhibits Janus kinase 3 (JAK3) and the tyrosine kinase expressed in hepatocellular carcinoma (TEC) family of kinases. Litfulo will be available in the next few weeks, Pfizer executives said in a press release.

The annual list price of Litfulo is $49,000, which is in line with other specialty dermatologic treatments, according to a Pfizer spokesperson. The company offers assistance through the Pfizer Dermatology Patient Access Program, and eligible patients will be able to get support with access to Litfulo, the spokesperson said.

FDA Rejects Higher Dose of Eylea

The FDA has issued a complete response letter (CRL) for a higher dose regimen of Eylea (aflibercept). Developed by Regeneron Pharmaceuticals and Bayer, Eylea is approved in a 2 mg dose to treat patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy (DR), as well as a 0.4 mg dose to treat retinopathy of prematurity. The companies were seeking an 8 mg dose to treat age-related macular degeneration, diabetic macular edema and diabetic retinopathy.

The CRL was issued related to an ongoing review of inspection findings at a third-party filer. Regulators did not identify any issues with the Eylea 8 mg clinical efficacy or safety, trial design, labeling or drug substance manufacturing, and no additional clinical data or trials have been requested.

At the same time, Regeneron released two-year data from a study evaluating the higher dose in patients with diabetic macular edema. In these patients, Eylea 2 mg is administered every four weeks for the first five injections and then every eight weeks. In the trial with the 8 mg dose, patients were given Eylea every 12 weeks or 16 weeks. The results indicated that patients maintained visual gains and safety was consistent with the original dose.

FDA Sets Action Date for Hemophilia B Gene Therapy

The FDA has accepted Pfizer’s biologics license application (BLA) for fidanacogene elaparvovec to treat adults with hemophilia B, and assigned a PDUFA date in the second quarter of 2024. In parallel, the European marketing authorization application (MAA) for fidanacogene elaparvovec has also been accepted and is under review by the European Medicines Agency (EMA).

Hemophilia is a rare genetic bleeding disorder caused by a deficiency in one of several blood clotting factors. Fidanacogene elaparvovec 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.

The submissions are based on efficacy and safety data from the phase 3, which met its primary endpoint of non-inferiority in the annualized bleeding rate (ABR) of total bleeds post-fidanacogene elaparvovec infusion versus prophylaxis regimen with Factor IX. Study results, which were reported in December 2022, also showed that key secondary endpoints demonstrated a 78% reduction in treated annualized bleeding rate and a 92% reduction in annualized infusion rate.

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