Sarepta Therapeutics Inc.’s Duchenne muscular dystrophy drug won approval from the U.S. FDA, capping months of regulatory uncertainty.

Pfizer acquired privately held gene therapy developer Bamboo Therapeutics in a deal worth up to $645 million to boost its presence in the treatment of rare diseases.

The FDA said companies could only charge patients for the cost of manufacturing experimental treatments used under compassionate grounds.

San Rafael, California-based BioMarin Pharmaceutical announced that it is abandoning its Kyndrisa (drisapersen) program for Duchenne muscular dystrophy (DMD).

The U.S. FDA deferred a highly anticipated decision on whether to approve Sarepta Therapeutics Inc.’s muscle-wasting disorder drug.

The U.S. FDA’s PCNSC voted against a recommendation for Sarepta Therapeutics’ eteplirsen for Duchenne Muscular Dystrophy (DMD).

Sarepta Therapeutics’ shares plunged more than 40 percent after U.S. FDA staff reviewers maintained their negative outlook on the company’s rare muscle wasting disorder drug.

March 22, 2016By Mark Terry, Breaking News Staff   Thirty-six experts in Duchenne Muscular Dystrophy (DMD) signed a letter to the U.S. Food and Drug Administration (FDA) urging the regulatory agency to approve eteplirsen, a drug developed by Cambridge, Mass.-based Sarepta Therapeutics (SRPT). Originally the FDA’s Peripheral and Central Nervous System Advisory Committee was […]

January 26, 2016By Mark Terry, Breaking News Staff   Cambridge, Mass.-based Akashi Therapeutics announced yesterday that after one of its patients in its clinical trial for HT-100 in Duchenne muscular dystrophy (DMD) developed serious, life-threatening health problems, it has halted the trial. The patient was receiving the highest dose in the HALO trial, 60µg/kd/d. […]

A panel of outside advisers to the U.S. Food and Drug Administration indicated that efficacy data from BioMarin Pharmaceutical Inc on its experimental drug for treating a muscle wastage disorder was not persuasive enough to warrant approval. The FDA panel did not officially vote on an approval of the drug, but 15 out of 17 […]