Keyword: PTC Therapeutics
The deal gives PTC ownership of a gene therapy treatment for rare neurotransmitter disease AADC deficiency that is set to go before the FDA next year.
Catabasis is looking to test its experimental Duchenne drug edasalonexent in a late-stage trial.
A panel of experts convened by the FDA has overwhelmingly knocked back PTC Therapeutics’ filing for approval of ataluren.
Akashi Therapeutics has been given the FDA go-ahead to restart tests for its Duchenne muscular dystrophy candidate HT-100 (delayed-release halofuginone), just over a year after the test was suspended after a death.
PTC Therapeutics is ending attempts to get its Duchenne drug ataluren onto the market in this disease setting after a key phase 3 trial flopped.
As Roche continues to try to hit all the bases in this rare disease, its partner PTC Therapeutics has started a midstage study for its candidate against certain forms of spinal muscular atrophy.
The FDA has denied the New Jersey company’s appeal of a refuse-to-file letter that dates back to February.
NICE has given PTC Therapeutics extra time to hash out a cost-effective price plan on Translarna.
The U.K.’s drug pricing watchdog initially rejected the drug a year ago, but today they announced that the drug will be covered after agreeing to an undisclosed discount.