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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 of Tazorac gel, and a gene therapy for a rare disease. Advisory committees vote down poziotinib for NSCLC and Pepaxto for multiple myeloma and give positive vote for microbiotic-based C. diff therapy. The agency has accepted sNDA for Tukysa for HER2 positive colorectal cancer.

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

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

This indication is approved under accelerated approval based on ORR and duration of response (DOR). Continued approval for this indication is contingent upon verification of clinical benefit in a confirmatory trial.

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

The two approvals are supported by data from the pivotal LIBRETTO-001 trial, which is the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor. The multicenter, open-label, multi-cohort study enrolled patients with locally advanced or metastatic RET-driven solid tumors, including NSCLC. Major efficacy outcomes 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 with magnetic resonance imaging (MRI). It is approved for use in adults and children aged 2 years and older to detect and visualize lesions with abnormal vascularity in the central nervous system (brain, spine and associated tissues) and the body (head and neck, thorax, abdomen, pelvis, and musculoskeletal system.

Elucirem will be marketed by Guerbet in the United States in bottle and pre-filled syringe form. It has been designed to allow it to be used at half the conventional dose of gadolinium compared with other non-specific gadolinium-based contrast agents. The approval is based on data from two phase 3 studies completed in March 2021, which demonstrated that Elucirem leads to non-inferior results in brain and body MRI at half the gadolinium dose of gadobutrol.

Bluebird bio’s gene therapy for CALD gets accelerated approval.

Shortly after approval of another gene therapy from bluebird bio, the FDA granted accelerated approval for the company’s Skysona (elivaldogene autotemcel), also known as eli-cel, to treat early, active cerebral adrenoleukodystrophy (CALD).

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

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

Last month, 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 of Tazorac gel.

The FDA has approved Cosette Pharmaceuticals’ abbreviated new drug application (ANDA) for the first generic versions of Almirall’s Tazorac (tazarotene) gel, 0.05% and 0.1%, which is used to treat psoriasis, acne, and sun damage to skin. The company has received 180 days 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 lowest retail price of the cream formulation is about $40 through mail order, according to GoodRx.

FDA advisory committee votes down Pepaxto for multiple myeloma

The FDA’s Oncologic Drugs Advisory Committee (ODAC) voted 14 to 2 on Sept. 22, 2022, against whether the benefit risk profile of Oncopeptides’ Pepaxto (melphalan flufenamide) was favorable in adult patients with relapsed or refractory multiple myeloma.

In materials released ahead of the meeting, the FDA indicated that the confirmatory trial (OCEAN) demonstrated a worse overall survival and failed to verify clinical benefit. The committee reviewed data from the OCEAN trial, which compared Peptaxto/dexamethasone with pomalidomide and dexamethasone in patients with relapsed or refractory multiple myeloma. Additionally, there were higher rates of deaths in Pepaxto arm than in the pomalidomide/dexamethasone arm. There were also higher rates of grade 3 and 4 adverse events in the Pepaxto arm.

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

FDA advisory committee votes against poziotinib for NSCLC.

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

The committee members agreed there is an unmet need in this patient population, but many said after the vote that poziotinib doesn’t appear to provide meaningful benefit beyond existing therapies, echoing concerns brought by the regulatory agency. The FDA expressed concerns about the toxicities associated with poziotinib, including high rates of treatment interruption and dose reduction, and very high rates of diarrhea, mucositis, and rash, as well as three fatal events of pneumonitis. In the ZENITH20 trial, poziotinib was poorly tolerated at the current proposed dosage (16 mg four times a day) with 57% of patients experiencing dose reductions and 85% of patients experiencing grade 3 to 4 adverse events.

FDA advisory committee gives positive vote for microbiota-based C. diff therapy.

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) issued a positive vote on Sept. 22, 2022, for Ferring Pharmaceuticals’ RBX2660 (now with the brand name Rebyota) to reduce recurrence of C. difficile infection (CDI) after antibiotic treatment. RBX2660 is microbiota-based live biotherapeutic that contains live microorganisms that are used as active substances. RBX2660 is a fecal microbiota transplantation therapy that was developed by Rebiotix, a Ferring company.

The committee voted 13 to 4 indicating that the data were adequate to support the effectiveness of RBX2660 to reduce the recurrence of C. difficile infection in adults 18 years of age and older following antibiotic treatment. The committee also voted 12 to 4 with one abstention that the data were adequate to support the safety of RBX2660.

C. diff is a serious disease 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 for HER2 positive colorectal cancer.

The FDA has accepted for priority review Seagen’s supplemental new drug application (sNDA) seeking accelerated approval for Tukysa (tucatinib). The company is seeking 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 a target action date of Jan. 19, 2023.

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 to treat adult patients with advanced unresectable or metastatic HER2-positive breast cancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting.

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