FDA Updates for Week of April 4, 2022

FDA approves novel therapy for schizophrenia and bipolar.

The FDA has approvedIgalmi (dexmedetomidine) for the acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. Developed by BioXcel Therapeutics, Igalmi is a sublingual film formulation of dexmedetomidine, a selective alpha-2 receptor agonist.

It is available in two doses: 120 mcg and 180 mcg. BioXcel expects to launch the therapy in the second quarter of 2022.

The approval is based on data from two phase 3 trials evaluating Igalmi for the acute treatment of agitation associated with schizophrenia (SERENITY I) or bipolar I or II disorder (SERENITY II).

In both trials, Igalmi met the primary endpoint, which was Positive and Negative Syndrome Scale-Excited Component (PEC) total score at two hours. This is an investigator-rated score for measuring agitation in patients that evaluates poor impulse control, tension, hostility, uncooperativeness, and excitement.

FDA grants accelerated approval for Vijoice to treat rare disorder.

The FDA has granted accelerated approval to Novartis’ Vijoice (alpelisib) for the treatment of adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-related overgrowth spectrum (PROS).

Vijoice, a kinase inhibitor, is the first FDA-approved treatment for PROS, a spectrum of rare conditions characterized by overgrowths and blood vessel anomalies impacting an estimated 14 people per million. The therapy works by inhibiting the PI3K pathway.

FDA approval was based on real-world evidence from EPIK-P1, a retrospective chart review study that showed patients treated with Vijoice experienced reduced target lesion volume and improvement in PROS-related symptoms and manifestations.

FDA extends PDUFA date for vutrisiran.

The FDA has extended the review timeline of Alnylam Pharmaceuticals’ new drug application (NDA) for vutrisiran, an investigational RNAi therapeutic to treat patients with transthyretin-mediated (ATTR) amyloidosis. The review has been extended three months to allow regulators to review newly added information related to the new secondary packaging and labeling facility. The new PDUFA date is July 14, 2022.

No inspection observations were directly related to vutrisiran and no additional clinical data have been requested by FDA, the company said in a statement.

A third-party secondary packaging and labeling facility Alnylam planned to use for the vutrisiran launch was recently inspected and the inspection requires classification for the FDA to take action on the vutrisiran NDA. To minimize delays to approval, Alnylam has identified a new facility to pack and label vutrisiran and submitted an amendment to the NDA for review by the FDA. No additional clinical data have been requested by the FDA.

Vutrisiran is administered subcutaneously every three months and treats both both hereditary ATTR (hATTR) amyloidosis and wild-type ATTR (wtATTR) amyloidosis.

ATTR amyloidosis is a protein misfolding disorder, according to the Cleveland Clinic. Transthyretin is a protein made by the liver that helps carry thyroid hormone and vitamin A in the blood. It can lead to amyloid deposits in the heart and/or the nerves and other organs and tissues

FDA accepts sBLA for Actemra for hospitalized COVID-19 patients.

The FDA has accepted the Genentech/Roche’s supplemental biologics license application (sBLA) and has granted priority review for Actemra (tocilizumab) intravenous for the treatment of COVID-19 in hospitalized adults who are receiving systemic corticosteroids and require supplemental oxygen.

If approved, Actemra would be the first U.S. FDA-approved immunomodulator for the treatment of COVID-19 in hospitalized patients.

The submission is based on results from four randomized, controlled studies that evaluated Actemra for the treatment of COVID-19 in more than 5,500 hospitalized patients. Altogether, the results of these four studies (EMPACTA, COVACTA, REMDACTA and RECOVERY) suggest that Actemra may improve outcomes in patients.

In June 2021, the FDA gave Actemra an emergency use authorization (EUA) and is currently approved for use in 16 countries around the world for defined patients hospitalized with severe or critical COVID-19.