FDA Updates for the Week of Sept. 19, 2022


The FDA approved a tumor-agnostic treatment for cancer, a lower-dose MRI contrast agent, the first generic version of Tazorac gel, and a gene therapy for a rare disease. Advisory committees reject poziotinib for NSCLC and Pepaxto for multiple myeloma and give a positive vote for microbiotic-based C. diff therapy. The Agency has accepted sNDA for Tukysa in HER2-positive colorectal cancer.

FDA grants accelerated approval of Retevmo for tumor-agnostic RET gene fusions.

The FDA has granted accelerated approval for Lilly’s Retevmo (selpercatinib) for adult patients with RET gene fusion. Tumor-agnostic data supporting the approval showed an overall response rate (ORR) of 44% across multiple tumor types in patients who progressed on or after prior systemic treatment.

This indication is being approved under accelerated approval based on ORR and duration of response (DOR). Further approval for this indication depends on the verification of the clinical benefit in a confirmatory study.

The FDA also gave Retevmo traditional approval for the treatment of adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a RET gene fusion. This extends the Retevmo label to patients with locally advanced disease and converts the accelerated approval for NSCLC to a traditional approval in May 2020.

The two approvals are supported by data from the pivotal LIBRETTO-001 study, the largest clinical study in patients with RET-related cancers treated with a RET inhibitor. The multi-center, open-label, multi-cohort study included patients with locally advanced or metastatic RET-driven solid tumors, including NSCLC. The main efficacy endpoints were ORR and DOR.

FDA Approves Lower-Dose Gadolinium-Based Contrast Agent.

The FDA has approved Guerbet’s Elucirem (gadopiclenol), a new macrocyclic gadolinium-based contrast agent for use in magnetic resonance imaging (MRI). It is approved for use in adults and children from 2 years of age to detect and visualize lesions with abnormal vascularity in the central nervous system (brain, spine and associated tissues) and body (head and neck, thorax, abdomen, pelvis, etc.). make musculoskeletal system.

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Elucirem is marketed by Guerbet in the United States in bottles and pre-filled syringes. It is designed to be used at half the traditional gadolinium dose compared to other non-specific gadolinium-based contrast agents. The approval is based on data from two Phase 3 studies completed in March 2021, which showed Elucirem resulted in no worse MRI results of the brain and body at half the gadolinium dose of gadobutrol.

Bluebird Bio’s Gene Therapy for CALD Receives Accelerated Approval.

Shortly after bluebird bio’s approval of another gene therapy, the FDA granted accelerated approval for Skysona (Elivaldogene Autotemcel), also known as Eli-Cel, for the treatment of early, active cerebral adrenoleukodystrophy (CALD).

The company also confirmed in a press release that the previous clinical hold on the eli-cel clinical development program has been lifted.

Bluebird has set Skysona’s wholesale cost at $3 million and expects the commercial product to be available through a limited number of qualified treatment centers in the United States by the end of this year.

Last month, the FDA also approved Bluebird Bio’s Zynteglo (Betibeglogene Autotemcel), also known as Beti-Cel, the first cell-based gene therapy to treat adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions.

FDA approves first generic Tazorac gel.

The FDA has approved Cosette Pharmaceuticals’ Abbreviated Marketing Authorization Application (ANDA) for the first generic versions of Almirall’s Tazorac (tazarotene) gel, 0.05% and 0.1%, used to treat psoriasis, acne and sun damage to the skin is used. The company has received a 180-day competitive generic therapy (CGT) exclusivity.

Tazarotene is a retinoid product related to vitamin A. Generics of the cream formulation are available from Cosette and Taro. The cream formulation’s lowest retail price is around $40 by mail order, according to GoodRx.

FDA advisory committee rejects Pepaxto for multiple myeloma

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 14-2 on September 22, 2022 on whether the benefit-risk profile of Pepaxto (melphalanflufenamide) is favorable from Oncopeptides in adult patients with relapsed or refractory multiple myeloma.

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In materials released prior to the meeting, the FDA noted that the confirmatory study (OCEAN) showed poorer overall survival and failed to verify clinical benefit. The committee reviewed data from the OCEAN study, which compared Peptaxto/dexamethasone to pomalidomide and dexamethasone in patients with relapsed or refractory multiple myeloma. In addition, there were higher death rates in the Pepaxto arm than in the pomalidomide/dexamethasone arm. Higher rates of grade 3 and 4 adverse events also occurred in the Pepaxto arm.

Almost all patients (99.6%) reported treatment-emergent adverse events, including serious events such as bleeding, infection, thrombocytopenia (low blood platelet counts) and neutropenia (low certain white blood cell counts). In addition, 53% of the 491 patients died during the studies, with most deaths occurring more than 30 days after the last Pepaxto dose.

FDA advisory committee votes against poziotinib for NSCLC.

On September 22, 2022, the FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 9-4 against whether the benefits of Spectrum Pharmaceuticals’ poziotinib outweigh the risks. The company sought accelerated approval of poziotinib for the treatment of non-small cell lung cancer (NSCLC) patients with HER2 exon 20 insertion mutations. Poziotinib is a kinase inhibitor with a suggested dosage of 16 mg four times a day.

Committee members agreed that there was an unmet need in this patient population, but many said after the vote that poziotinib does not appear to offer any meaningful benefit beyond existing therapies, reflecting concerns raised by the regulator. The FDA expressed concern about toxicities associated with poziotinib, including frequent treatment interruptions and dose reductions, very high rates of diarrhea, mucositis, and rash, and three fatal pneumonitis events. In the ZENITH20 study, poziotinib at the currently proposed dose (16 mg qid) was poorly tolerated, with 57% of patients experiencing dose reductions and 85% of patients experiencing Grade 3-4 adverse events.

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FDA Advisory Committee gives positive vote for microbiota-based C. diff Therapy.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) gave a positive vote for Ferring Pharmaceuticals’ RBX2660 (now branded Rebyota) on September 22, 2022 to reduce the recurrence of C. difficile Infection (CDI) after antibiotic treatment. RBX2660 is a microbiota-based live biotherapeutic containing live microorganisms used as active ingredients. RBX2660 is a fecal microbiota transplant therapy developed by Rebiotix, a Ferring company.

The committee voted 13 to 4, indicating the data were sufficient to establish the effectiveness of RBX2660 in reducing the recurrence of C. difficile Infection in adults 18 years and older after antibiotic treatment. The committee also voted 12 to 4 with 1 abstention that the data was adequate to support the security of the RBX2660.

C. diff is a serious illness that causes severe diarrhea, fever, stomach tenderness or pain, loss of appetite, nausea, and colitis. It has been estimated that up to 35% of cases recur after initial diagnosis and people who have had a recurrence are at significantly higher risk of further infection.

FDA accepts sNDA for Tukysa in HER2-positive colorectal cancer.

The FDA has accepted Seagen’s Supplemental New Drug Application (sNDA) for accelerated approval of Tukysa (tucatinib) for priority review. The Company is targeting an indication in combination with trastuzumab for adult patients with HER2-positive colorectal cancer who have received at least one prior treatment regimen for unresectable or metastatic disease. The agency has set January 19, 2023 as the target date.

Tukysa is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. The FDA approved Tukysa in April 2020 in combination with trastuzumab and capecitabine for the treatment of adult patients with advanced, unresectable, or metastatic HER2-positive breast cancer, including patients with brain metastases who have previously received one or more anti-HER2-based treatments in metastatic treatment have received attitude.



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