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FDA Updates for the Week of Oct. 31, 2022


The FDA has approved a hepatitis B therapy for adolescents and an oral MEK inhibitor to treat blood cancers. The regulatory agency has granted priority review for an adult RSV vaccine but extended the review for a Pompe disease therapy. Additionally, CytoDyn has withdrawn its BLA for an HIV therapy.

FDA approves hepatitis B therapy for adolescents.

The FDA has approved the supplemental new drug application (sNDA) for Gilead Sciences’ Vemlidy (tenofovir alafenamide) to treat chronic hepatitis B virus (HBV) infection in pediatric patients 12 years of age and older with compensated or asymptomatic liver disease.

Vemlidy is a targeted prodrug of tenofovir that was approved by the FDA in 2016 as a once-daily treatment for adults with chronic hepatitis B virus infection with compensated liver disease. It is recommended as a preferred or first-line treatment for adults with chronic HBV with compensated liver disease.

The average retail price for Vemlidy is $2,034.78 for 30 tablets, with the lowest retail price of $1,285.33, according to GoodRx.

Approves melanoma drug to treat blood cancers.

The FDA okayed Cotellic (cobimetinib) to treat adult patients with the family of blood diseases known as histiocytic neoplasms (HN). The diseases include Erdheim-Chester disease, Rosai-Dorfman disease, and Langerhans cell histiocytosis.

Cotellic is an oral inhibitor of MEK1 and MEK2 that currently approved to treat melanoma, Memorial Sloan Kettering Cancer Center (MSK) said in a news release. Before now, available treatment options for most adults with histiocytic neoplasms were limited and associated with poor long-term tolerance, according to MSK.

The approval was granted based on data collected by MSK in collaboration with Genentech, a member of the Roche Group, from a single-institution phase 2 trial of single-agent Cotellic for adults with histiocytic disorders. “A rarity in the world of cancer research, the clinical trial that led to the approval was conducted solely at MSK,” the organization said.

FDA grants priority review for adult RSV vaccine.

The FDA has accepted a biologics license application (BLA) and granted priority review for GSK’s vaccine for respiratory syncytial virus (RSV) for older adults. The Prescription Drug User Fee Act date is May 3, 2023. If approved, GSK’s RSV older adult vaccine candidate has the potential to be the first vaccine available to help protect adults aged 60 years and older from lower respiratory tract disease caused by RSV infection.

RSV is a common contagious virus affecting the lungs. There is currently no vaccine or specific treatment available for adults. Older adults are at high risk for severe disease due to age-related decline in immunity and underlying conditions. RSV can exacerbate conditions, including chronic obstructive pulmonary disease (COPD), asthma and chronic heart failure and can lead to severe outcomes, such as pneumonia, hospitalization, and death.

The BLA is based on positive data from a prespecified interim analysis of the pivotal AReSVi-006 (Adult Respiratory Syncytial Virus) phase 3 trial, which showed high overall vaccine efficacy against RSV lower respiratory tract disease in adults aged 60 years and older.

FDA Again Extends Review for Pompe Disease Therapy

The FDA has deferred action on Amicus Therapeutics’ biologics license application (BLA) for cipaglucosidase alfa, the biologic component of AT-GAA. Due to restrictions on travel related to COVID-19, the FDA was unable to conduct the required inspection of the WuXi Biologics manufacturing site in China during the review cycle.

AT-GAA is a therapeutic to treat patients with Pompe disease, a genetic lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA) that leads to severe muscle weakness that worsens over time. AT-GAA contains two components: cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme, and miglustat, a stabilizer of cipaglucosidase alfa. Amicus officials expect the FDA to approve the two components of AT-GAA, including the BLA for cipaglucosidase alfa and a new drug application for miglustat, together.

This is second time action this therapeutic has been delay. In May 2022, the FDA had extended the review of this BLA by 90 days to allow additional time to review information submitted by the company as part of its ongoing reviews.

CytoDyn withdraws BLA for HIV therapy.

CytoDyn has voluntarily withdrawn its pending biologics license application (BLA) for leronlimab, a humanized monoclonal that targets the CCR5 receptor, to treat people with HIV who are multi-drug resistant (MDR). The withdrawal is related to the quality of the data collection and monitoring of the pivotal clinical trials by the clinical research organization (CRO) contracted to manage the trials, the company said in a press release.

The BLA was based on data from phase 2b/3 placebo-controlled assessing leronlimab, in combination with a highly active antiretroviral therapy (HAART), to treat highly treatment-experienced HIV patients. In July 2020, the company received a refusal to file letter from the FDA regarding its BLA submission for leronlimab. The FDA cited deficiencies related to data presentation and related analyses, and also requested clarifications regarding the manufacturing processes.

In November 2021, CytoDyn resubmitted the non-clinical and chemistry, manufacturing, and controls sections of the BLA. The contract manufacturing company, however, at that time refused to release the clinical data, and in March 2022, the FDA had placed a partial clinical hold on CytoDyn’s HIV program.

Company officials believe the data it currently possesses is sufficient to complete and submit its responses to the FDA to seek the removal of the clinical hold placed on the company's HIV program. CytoDyn plans to publish the safety and efficacy data in which it met its primary endpoint, in its phase 2b/3 placebo-controlled trial for the HIV-MDR population

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