1. Aug 28, 2020

FDA Roundup: This week's FDA approvals (August 22-28)

FDA approves liquid biopsy with multiple companion diagnostic indications for advanced cancer
The U.S. Food and Drug Administration has approved FoundationOne Liquid CDx, a comprehensive pan-tumor liquid biopsy test, according to a press release.

We believe that cancer patients and their physicians deserve the highest quality genomic testing to make informed decisions about personalized treatment,” said Brian Alexander, MD, MPH, Chief Medical Officer at Foundation Medicine, in a press release. “Created from our scientific expertise and pioneering spirit, FoundationOne Liquid CDx underscores our commitment to advance patient care across all cancer types by bringing forward multiple FDA-approved comprehensive genomic profiling options that are increasingly essential for high-quality cancer care. We are seeking additional companion diagnostic claims for FoundationOne Liquid CDx, which, if approved, would further enhance utility of the test in clinical practice. Additionally, this test is an important tool for the acceleration of drug development and for understanding mechanisms of resistance.”

Read the full press release here.

FDA grants rare pediatric disease designation to sickle cell disease therapy
The U.S. Food and Drug Administration has granted rare pediatric disease designation to EDIT-301, an experimental, autologous cell medicine for sickle cell disease. Editas Medicine, Inc plans to file an investigational new drug application for EDIT-301 by the end of 2020, according to a company press release.

“The Editas team has a bold vision to unlock the potential of CRISPR to design and develop game-changing medicines. We are making tremendous progress towards this vision with the continued development of EDIT-301, a potentially transformative medicine for the treatment of sickle cell disease, and we are pleased to receive Rare Pediatric Disease designation from the FDA for this program,” said Cynthia Collins, Chief Executive Officer, Editas Medicine in a press release.  “We know patients are counting on us, and this designation is a significant milestone for the program that highlights the serious, life-threatening manifestations of sickle cell disease.”

Read the full press release here

FDA removes boxed warning about amputations on canagliflozin label
The U.S. Food and Drug Administration removed the boxed warning about amputation risk from the diabetes medicine canagliflozin after reviewing new data, according to a press release.

New data from 3 clinical trials demonstrated additional heart- and kidney-related benefits and led to new approved uses.

Collectively, these newly identified effects of canagliflozin on heart and kidney disease show significantly enhanced benefit of this medicine.  Safety information from recent clinical trials also suggests that the risk of amputation, while still increased with canagliflozin, is lower than previously described, particularly when appropriately monitored.  Based upon these considerations, FDA has concluded that the boxed warning should be removed,” read the FDA press release.

Read more here

FDA approves ofatumumab for relapsing multiple sclerosis
The U.S. Food and Drug Administration has approved ofatumumab (Kesimpta; Novartis) injection for subcutaneous use for the treatment of patients with relapsing multiple sclerosis, according to a press release.

Ofatumumab is the only self-administered, targeted B-cell therapy for patients with relapsing multiple sclerosis.

Approval is based on results from the Phase III ASCLEPIOS I and II studies. In the studies, ofatumumab showed superiority versus teriflunomide in significantly reducing the annualized relapse rate, 3-month confirmed disability progression, and the number of gadolinium-enhancing T1 and new or enlarging T2 lesions.

“This approval is wonderful news for patients with relapsing multiple sclerosis. In the key clinical studies, this breakthrough treatment produced a profound reduction in new brain lesions, reducing relapses and slowing underlying disease progression1,” said Professor Stephen L. Hauser, Director of the UCSF Weill Institute for Neurosciences and co-chair of the steering committee for the ASCLEPIOS I and II studies, in a press release. “Through its favorable safety profile and well-tolerated monthly injection regimen, patients can self-administer the treatment at home, avoiding visits to the infusion center.”

Read the full press release here