Topic: spinal muscular atrophy
The clinical success comes months before the FDA is set to decide whether to approve the survival motor neuron-2 splicing modifier.
A one-time medicine intended to cure patients, Zolgensma scored an endorsement from cost watchdogs at ICER despite the record price.
The results tee Roche up to file for approval to compete with Biogen and Novartis in the increasingly crowded spinal muscular atrophy market.
6. Michael Ehlers, Biogen
Following its $15 million series A round earlier this year, BillionToOne has launched its first product for commercial and clinical use.
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.
Blocking a pathway that causes healing cells to turn bad could offer a new way to treat neurological disorders.
Biogen has bolted on a couple of early-stage drug candidates for neuromuscular diseases via a $535 million deal with California startup AliveGen.
The Swiss pharma is walking away from olesoxime after running into “many difficulties in developing” the oral mitochondria-boosting drug.