FDA Updates for Week of May 13: First Bispecific Antibody for Solid Tumor


The FDA has approved a new type of bispecific antibody to treat small cell lung cancer and an additional indication for Breyanzi for patients with follicular lymphoma. The agency has set review date for gene therapy for enzyme deficiency. In addition, Biogen have Eisai hve begun a rolling submission of subcutaneous Leqembi for Alzheimer’s disease.

FDA Approves Bispecific Antibody for Small Cell Lung Cancer

The FDA has granted accelerated approval for Amgen’s Imdelltra (tarlatamab-dlle) to treat adult patients with extensive-stage small cell lung cancer (ES-SCLC). It is indicated for patients with disease progression on or after platinum-based chemotherapy.

About 10% to 15% of all lung cancers are small cell, according to the American Cancer Society. Small cell lung cancer is an aggressive solid tumor with a median survival of about 12 months following initial therapy and a 3% five-year relative survival rate.

Imdelltra is a first-in-class delta-like ligand 3 (DLL3) targeting Bispecific T-cell Engager (BiTE) therapy and the first bispecific antibody for extensive-stage small cell lung cancer. Imdelltra targets CD3 on T cells and DLL3 on small cell lung cancer cells.

Imdelltra will have a list price of $31,500 for the first cycle and $30,000 for the second cycle, according an Amgen spokesperson. Amgen will host a webcast call on Monday, May 20, 2024 at 1:00 p.m. PT (4:00 p.m. ET).

The accelerated approval was based on the response rate and duration of response seen in the phase 2 DeLLphi-301 clinical trial, which studied 220 patients with advanced small cell lung cancer. Imdelltra demonstrated an objective response rate of 40% and a median duration of response 9.7 months. The median overall survival was 14.3 months.

FDA Approves Breyanzi for Follicular Lymphoma

The FDA has granted accelerated approval for Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel; liso-cel) to treat adult patients with relapsed or refractory follicular lymphoma have received two or more prior lines of systemic therapy.

Follicular lymphoma is the second most common, slow-growing form of non-Hodgkin lymphoma (NHL), accounting for 20% to 30% of all NHL cases. It develops when white blood cells cluster together to form lumps in a person’s lymph nodes or organs.

Breyanzi is a CD19-directed chimeric antigen receptor (CAR) T cell therapy that is already approved to treat adult patients with large B-cell lymphoma and with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

Breyanzi is derived from a patient’s own T cells that have been engineered to target CD19 B cells. Breyanzi includes a box warning about the risk of cytokine release syndrome, a large release of cytokines into the blood, which triggers overreaction of the immune system and neurological toxicities. The list price for Breyanzi is $487,477.43, according to Drugs.com. For full year 2023, Breyanzi generated revenue of $364 million.

Breyanzi is also now included in the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) for B-cell Lymphomas as a Category 2A recommendation for third-line and subsequent therapy for relapsed or refractory FL.

FDA Sets Review Date for Gene Therapy for Enzyme Deficiency

The FDA has accepted the biologics license application (BLA) for Upstaza (eladocagene exuparvovec). The application has been granted priority review with a target regulatory action date of Nov. 13, 2024.

Developed by PTC Therapeutics, Upstaza is a one-time gene therapy to treat patients 18 months and older with aromatic L-amino acid decarboxylase (AADC) deficiency, a fatal, rare genetic disorder that typically causes severe disability. It leads to metabolic disturbance of dopamine, norepinephrine, epinephrine, and serotonin. Children with AADC deficiency can experience seizure-like oculogyric crises (involving the eyes), frequent vomiting, behavioral problems, and difficulty sleeping.

AADC deficiency is caused by mutations of the DOPA decarboxylase (DDC) gene in which the AADC enzyme cannot perform its function in the formation neurotransmitters epinephrine and norepinephrine, dopamine and serotonin.

Upstaza is a recombinant adeno-associated virus serotype 2 (AAV2)-based gene therapy, containing the human DDC gene. It is designed to deliver a functioning DDC gene directly into the putamen, the part of the brain involved in learning and motor control. Administration of Upstaza is designed to increase the AADC enzyme and restore dopamine production.

Biogen, Eisai Begin Submission of Subcutaneous Leqembi

Biogen and Eisai have begun a rolling submission of a biologics license application (BLA) to the FDA for a subcutaneous autoinjector for weekly dosing of Leqembi (lecanemab-irmb). The FDA has granted this a fast track designation. If approved, the Leqembi autoinjector could be used to administer the medication at home or at medical facilities.

An intravenous formulation of Leqembi is indicated to treat adults with early Alzheimer’s disease (AD). Leqembi has been on the market since January 2023 when it received an accelerated approval. It has a list price of $26,500 a year. The IV formulation of Leqembi received full approval in July 2023 based on phase 3 data from Eisai’s global Clarity AD clinical trial. In March 2024, Biogen and Eisai announced they had submitted a supplemental application for a monthly maintenance dose of the IV formulation of Leqembi.

Weekly subcutaneous administration of Leqembi removed 14% more amyloid plaque in patients with Alzheimer’s disease than the biweekly intravenous formulation, according to data released at the annual Clinical Trials on Alzheimer’s Disease (CTAD) conference in October 2023. Additionally, 60% of the patients with early stage Alzheimer’s disease and who have low levels of the protein tau achieved cognitive improvement when treated with Leqembi.

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