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FDA Updates for Week of May 9, 2022


In COVID-19 news, the FDA has approved Olumiant for new COVID-19 indication, but had limited Janssen’s COVID-19 vaccine. The agency has launched a new program for rare disease drug development and approved a new oral form of ALS therapy. Regulators have also extended the review time for both a new Pompe disease therapy and the sNDA for Myfembree for endometriosis. Additionally, Eisai has completed its submission of lecanemab for Alzheimer’s disease.

FDA launches new rare disease program.

The FDA has launched a new program that is aimed to increase the development of drugs for people with rare diseases. Called Accelerating Rare Disease Cures (ARC), the new program is managed by Center for Drug Evaluation and Research’s (CDER) Rare Diseases Team and will provide coordination across the agency’s efforts in this area.

This new effort continues the regulator agencies effort to provide support for the development of rare diseases. One effort, developed in collaboration with the Critical Path Institute, is the Rare Disease Cures Accelerator-Data and Analytics Platform. The platform provides a database designed to promote the secure sharing of existing patient-level data and encourage the standardization of new data collection.

FDA approves Olumiant for new COVID-19 indication.

The FDA has approved a new indication for Eli Lilly and Incyte’s Olumiant (baricitinib) for COVID-19 in hospitalized adults requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO).

Olumiant is the first immunomodulatory treatment for COVID-19 to receive FDA approval, the FDA said in a news release.

The agency first issued an emergency use authorization (EUA) in November 2020 for Olumiant in combination with remdesivir to treat COVID-19 in hospitalized adults and pediatric patients. In July 2021, the FDA revised the EUA to authorize Olumiant as a stand-alone treatment.

Olumiant remains under EUA status for hospitalized pediatric patients 2 to less than 18 years of age requiring supplemental oxygen, non-invasive or invasive mechanical ventilation, or ECMO.

FDA limits Janssen’s COVID-19 vaccine.

Due to the risk of blood clots, the FDA is limiting the authorized use of the Janssen COVID-19 vaccine. Now, the use of the vaccine is limited to people 18 years of age and older for whom other authorized or approved COVID-19 vaccines are not accessible or clinically appropriate, the FDA said in a statement.

After conducting an updated analysis, evaluation and investigation of reported cases, the FDA has determined that the risk of thrombosis with thrombocytopenia syndrome (TTS), a rare syndrome of potentially life-threatening blood clots in combination with low levels of blood platelets with onset of symptoms.

FDA approves oral form of ALS therapy.

The FDA has approved Radicava ORS (edaravone) oral suspension for the treatment of adults with amyotrophic lateral sclerosis (ALS). Radicava ORS, developed by Mitsubishi Tanabe Pharma America, is an orally administered version of Radicava, which was originally approved in 2017 as an intravenous (IV) infusion to treat ALS.

Radicava ORS can be taken orally or administered through a feeding tube. It has the same dosing regimen as Radicava—an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period and subsequent treatment cycles consisting of daily dosing for 10 out of 14-day periods, followed by 14-day drug-free periods.

FDA extends review time for Pompe disease therapy.

The FDA has extended the review by 90 days for Amicus Therapeutics’ AT-GAA, 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.

The company had submitted two applications for this therapy, one for each component. This includes a biologics license application (BLA) for cipaglucosidase alfa, which has been extended to Oct. 29, 2022, and a new drug application (NDA) for miglustat, which has been extended to Aug. 29, 2022.

The FDA extended the PDUFA dates to allow additional time to review information submitted by the company as part of its ongoing reviews. The extension of the review timeline was not related to requests for any additional clinical data.

FDA extends review of sNDA for Myfembree

The FDA has extended the review of the supplemental new drug application (sNDA) for Myfembree (relugolix, estradiol, and norethindrone acetate) for the management of moderate-to-severe pain associated with endometriosis. Regulators want additional time to review information they had requested from the companies regarding bone mineral density. The extended Prescription Drug User Fee Act (PDUFA) goal date is August 6, 2022.

Developed by Myovant Sciences and Pfizer, Myfembree was approved in the United States in May 2021 for the management of heavy menstrual bleeding associated with uterine fibroids in premenopausal women. Data to support this indication was based on the phase 3 LIBERTY 1 and LIBERTY 2 trials, which were published in the New England Journal of Medicine.

In April 2022, regulators had notified the companies that there were deficiencies in the application for endometriosis indication.

Eisai completes submission of lecanemab for Alzheimer’s disease.

Eisai has completed the rolling submission to FDA for an accelerated approval of lecanemab, another monoclonal antibody that targets beta amyloid, to treat mild cognitive impairment in patients with early Alzheimer’s disease. Eisai has requested priority review.

Eisai is continuing with a confirmatory phase 3 study of lecanemab with almost 1,800 patients enrolled and with results expected in the fall of this year. The FDA has agreed that this trial could be used as the confirmatory study to verify clinical benefit.

The BLA submission for lecanemab is based on clinical, biomarker and safety data from the proof-of-concept phase 2b in 856 people with early Alzheimer’s disease with confirmed presence of amyloid pathology, biomarker and safety data from an open-label extension study, and blinded safety data from the confirmatory phase 3 study.

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