Keyword: PTC Therapeutics
The clinical success comes months before the FDA is set to decide whether to approve the survival motor neuron-2 splicing modifier.
Roche and partner PTC Therapeutics are the latest companies to show their hand in improving kids’ ability to move with spinal muscular atrophy.
The interim readout showed that two-fifths of infants who received PTC and Roche’s oral SMA drug were sitting eight months after starting treatment.
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.