Rare diseases
The Phase III, open-label extension study suggests EPX-100 has a “positive” risk/benefit profile for the treatment of Dravet syndrome, analysts at H.C. Wainwright & Co. said Tuesday.
If approved, Ascendis’ TransCon CNP would become the second therapy for achondroplasia, challenging BioMarin’s Voxzogo.
This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question whether they will be enough to net their developers a significant share of the entrenched market.
Since July, several biotechs have been forced to pivot as previous agreements with the FDA around evidence required for approval were reversed, a phenomenon that, according to experts, could portend a more restrictive regulator.
The regulator has received reports that a group of patients treated with Adzynma had neutralizing antibodies against the protein the therapy replaces.
Korro Bio is moving back to square one as a preclinical biotech after the failure of KRRO-110 in alpha-1 antitrypsin deficiency. The company’s stock is down 80% on all the news.
The plausible mechanism pathway “could accelerate gene therapy/editing development,” analysts at William Blair said Thursday, while adding that additional clarity is needed.
One of the FDA’s potential approvals this month could break an existing monopoly in the treatment space for a rare growth disorder.
According to reporting from Reuters, reviewers at the agency pointed to an inability to differentiate from placebo to justify rejecting the drug, but an FDA office director approved the drug anyway.
Biohaven is proposing troriluzole for the treatment of spinocerebellar ataxia, a group of rare, genetic diseases that lead to the progressive loss of control over movement.
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