BioMarin Gets Second PKU Approval, Anticipates Slow Ramp-Up For Palynziq
Analysts say black box warning of anaphylaxis risk and REMS were anticipated. Company plans a gradual rollout of Palynziq, focusing first on Phase III clinical trial sites and patients.
You may also be interested in...
The number of US drug approvals in 2018 was striking, but most of the novel products launched last year are for rare diseases or niche cancers, meaning commercial success may be in the eye of the beholder.
Opinions on whether new products for the treatment of conditions such as psoriasis, coagulation reversal, flu and diabetes should be granted an EU marketing authorization are due this week as the EMA’s key scientific committee, the CHMP, meets for the last time in London..
During BioMarin’s earnings call Feb. 21, CEO Bienaime indicated the company may be looking to price its valoctocogene roxaparvovec gene therapy at $2m-$3m, as it would represent a savings over the annual cost of infused clotting factor therapy.