FDA approves drug to treat ALS

FDAThe U.S. Food and Drug Administration today approved Radicava (edaravone) to treat patients with amyotrophic lateral sclerosis (ALS), commonly referred to as Lou Gehrig's disease. ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. Voluntary muscles produce movements such as chewing, walking, breathing and talking. The nerves lose the ability to activate specific muscles, which causes the muscles to become weak and leads to paralysis. ALS is progressive, meaning it gets worse over time. The Centers for Disease Control and Prevention estimates that approximately 12,000-15,000 Americans have ALS. Most people with ALS die from respiratory failure, usually within three to five years from when the symptoms first appear.

"After learning about the use of edaravone to treat ALS in Japan, we rapidly engaged with the drug developer about filing a marketing application in the United States," said Eric Bastings, M.D., deputy director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. "This is the first new treatment approved by the FDA for ALS in many years, and we are pleased that people with ALS will now have an additional option."

Radicava is an intravenous infusion given by a health care professional. It is administered with an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period. Subsequent treatment cycles consist of dosing on 10 of 14 days, followed by 14 days drug-free.

The efficacy of edaravone for the treatment of ALS was demonstrated in a six-month clinical trial conducted in Japan. In the trial, 137 participants were randomized to receive edaravone or placebo. At Week 24, individuals receiving edaravone declined less on a clinical assessment of daily functioning compared to those receiving a placebo.

The most common adverse reactions reported by clinical trial participants receiving edaravone were bruising (contusion) and gait disturbance.

Radicava is also associated with serious risks that require immediate medical care, such as hives, swelling, or shortness of breath, and allergic reactions to sodium bisulfite, an ingredient in the drug. Sodium bisulfite may cause anaphylactic symptoms that can be life-threatening in people with sulfite sensitivity.

The FDA granted this drug orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted approval of Radicava to Mitsubishi Tanabe Pharma America, Inc.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency is also responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

Most Popular Now

FDA approves first biosimilar for the treatment of…

The U.S. Food and Drug Administration today approved Mvasi (bevacizumab-awwb) as a biosimilar to Avastin (bevacizumab) for the treatment of multiple types of cancer. Mvas...

Merck set to join forces with Project Data Sphere …

Merck, a leading science and technology company has announced that it will enter into a strategic collaboration with Project Data Sphere LLC, an independent, not-for-prof...

FDA approval brings first gene therapy to the Unit…

The U.S. Food and Drug Administration issued a historic action today making the first gene therapy available in the United States, ushering in a new approach to the treat...

Novartis appoints Bertrand Bodson as Chief Digital…

Novartis announced today that Bertrand Bodson, Chief Digital and Marketing Officer for Sainsbury's Argos, has been appointed to the new role of Chief Digital Officer, rep...

Boehringer Ingelheim Pharmaceuticals, Inc. receive…

Boehringer Ingelheim Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) approved CyltezoTM, a biosimilar to Humira®*, in a pre-filled sy...

Asthma medicine halves risk of Parkinson's

Parkinson's disease is a chronic disease with unknown causes. The disease destroys the brain cells that control body movements. Shivering, stiff arms and legs and poor co...

This is how belly fat could increase your cancer r…

It's been well established that obesity is a contributor to cancer risk, but how it actually causes cancer is still a question that hasn't been fully explained. A new Mic...

Amgen and Humana partner for improved health outco…

Two of the nation's leading health organizations, health and well-being company Humana Inc. (NYSE: HUM) and biotechnology company Amgen (NASDAQ:AMGN), have teamed up to i...

Tezepelumab significantly reduced asthma exacerbat…

AstraZeneca and Amgen Inc. (Amgen) announce results from the PATHWAY Phase IIb trial of tezepelumab that showed a significant reduction in the annual asthma exacerbation ...

Sanofi and Regeneron announce that cemiplimab (REG…

Sanofi and Regeneron Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to cemipli...

Boehringer Ingelheim initiates Phase IIa study of …

Boehringer Ingelheim and pharmaceutical company Pharmaxis (ASX: PXS) announce that Boehringer Ingelheim has initiated a European and North American Phase IIa trial in NAS...

Extended treatment with Brilinta reduces risk of c…

AstraZeneca today announced results from a new sub-analysis of data from the Phase III PEGASUS-TIMI 54 trial demonstrating a 29% risk reduction in CV death (p=0.0041) fro...

Pharmaceutical Companies

[ A | B | C | D | E | F | G | H | I | J | K | L | M | N | O | P | Q | R | S | T | U | V | W | X | Z ]