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FDA Updates for Week of March 11: First CAR-T for CLL/SLL and First NASH Treatment

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The FDA this week approved a few firsts: the first treatment for NASH and the first CAR-T cell therapy for CLL/SLL. Other approvals include Tevimbra for esophageal cancer; Livmarli for second liver disease indication; and Praluent for children with genetic form of high cholesterol. The FDA also issued a CRL for monthly MS drug. Additionally, Celltrion submitted an application for an interchangeable Xolair biosimilar.

FDA Approves Breyanzi for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma

The FDA has granted accelerated approval of Breyanzi (lisocabtagene maraleucel; liso-cel), to treat adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). It is indicated for those who have received at least two prior lines of therapy.

CLL and SLL are among the most common types of B-cell lymphoma. Treatments primarily consist of targeted therapies including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor.

Developed by Bristol Myers Squibb, Breyanzi is a CD19-directed CAR T cell therapy. It is already approved to treat adult patients with large B-cell lymphoma and follicular lymphoma who have refractory disease or who have relapsed. The list price of Breyanzi is $487,477.43. For full year 2023, Breyanzi generated revenue of $364 million.

FDA Approves Rezdiffra, First Drug for NASH

The FDA announced that it had granted accelerated approved for Rezdiffra (resmetirom), the first drug approved for noncirrhotic non-alcoholic steatohepatitis (NASH).

During an investors conference call, Madrigal CEO Bill Sibold, MBA, said the new drug would be priced at the wholesale acquisition cost (WAC) price of $47,400 per year. That is at the high end of the $39,600-$50,100 range that the Institute for Clinical and Economic Review, an independent cost-effectiveness research group, set for the drug if it were to meet commonly accepted cost-effectiveness thresholds.

FDA Approves Tevimbra for Esophageal Cancer

The FDA has approved Tevimbra (tislelizumab-jsgr) as monotherapy to treat adult patients with unresectable or metastatic esophageal squamous cell carcinoma (ESCC). It is indicated to be used after prior systemic chemotherapy that did not include a PD-(L)1 inhibitor.

Developed by BeiGene, Tevimbra is humanized IgG4 anti-PD-1 monoclonal antibody designed to aid the body’s immune cells to detect and fight tumors. It will be available in the United States in the second half of 2024. A company spokesperson said pricing will be available closer to launch. BeiGene is currently in discussions with payers regarding coverage.

The approval is based on the RATIONALE 302 trial, which met its primary end point in the intention-to-treat (ITT) population. In the ITT population, the median overall survival (OS) in the Tevimbra arm was 8.6 months compared with 6.3 months in the chemotherapy arm.

FDA Approves Livmarli for Second Liver Disease Indication

The FDA has approved an additional indication for Livmarli (maralixibat) oral solution: to treat cholestatic pruritus (itch) in patients 5 years of age and older with progressive familial intrahepatic cholestasis (PFIC).

Progressive familial intrahepatic cholestasis (PFIC) is a rare genetic disorder that causes progressive liver disease. In people with PFIC, liver cells are less able to secrete bile.

Developed by Mirum Pharmaceuticals, Livmarli was approved by the FDA in 2021 to treat cholestatic pruritus in Alagille syndrome (ALGS) in children 1 year of age and older. In March 2023, the indication was expanded to include infants three months and older.

The cost for Livmarli oral solution (9.5 mg/mL) is about $56,240 for a supply of 30 milliliters, according to Drugs.com. In 2023, Livmarli generated global net sales of $141.8 million, an 89% growth over 2022 net sales.

The approval for children with PFIC is based on data from the phase 3 MARCH study, which enrolled 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status.

FDA Approves Praluent for Children with Genetic Form of High Cholesterol

The FDA has approved Regeneron’s Praluent (alirocumab) to treat children with a genetic form of high cholesterol. This approval extends Praluent’s indication to treat children age 8 years and older with heterozygous familial hypercholesterolemia (HeFH).

Familial hypercholesterolemia (FH) is an inherited condition caused by mutations in one of several genes that control how the body processes cholesterol.

Developed by Regeneron and Sanofi, Praluent inhibits the binding of PCSK9 to the LDL receptor and thereby increases the number of available low-density lipoprotein (LDL) receptors on the surface of liver cells to clear LDL and lower LDL cholesterol levels in the blood. The wholesale acquisition cost of Praluent is $506.48 for two single-dose pens.

The approval is based on a phase 3 trial that enrolled patients aged 8 to 17 with HeFH, who had LDL-C levels of 130mg/dL or greater and were already being treated with lipid-lowering medications. I

FDA Issues CRL for Monthly MS Drug

The FDA has issued a complete response letter (CRL) for the new drug application (NDA) for GA Depot 40 mg to treat patients with relapsing forms multiple sclerosis. No information has been provided for why the FDA would not approve the application.

Developed by Viatris and Mapi Pharma, GA Depot is a long-acting version of glatiramer acetate, which is available as generics and as the branded therapy Copaxone. Glatiramer acetate stops the immune system from attacking myelin. Available glatiramer acetate therapies involve injections three times a week, whereas GA Depot would be administered once every four weeks.

Celltrion Submits Application for Interchangeable Xolair Biosimilar

Celltrion USA has submitted a biologics license application (BLA) for CT-P39, an interchangeable biosimilar that references Genentech’s Xolair (omalizumab).

The BLA submission includes results from a global phase 3 clinical trial designed to evaluate the efficacy, safety, and pharmacokinetics of CT-P39 compared with the reference product Xolair in patients with chronic spontaneous urticaria (CSU) up to Week 40. The application includes the same indication as Xolair, including asthma, chronic rhinosinusitis with nasal polyps (CRSwNP), chronic spontaneous urticaria and its recently approved indication IgE-mediated food allergy.

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