BioMarin submits NDA for controversial achondroplasia drug; Cell and gene therapy boom sparks €49M buyout
BioMarin is plowing ahead with its achondroplasia candidate, submitting an NDA to the FDA for vosoritide Thursday afternoon.
If approved, the drug would be a once-daily injection for children with the condition, which is the most common cause of dwarfism. The application follows the release of topline Phase III trial results back in December showing vosoritide improved annual growth by 1.6 cm against placebo in children between the ages of 5 and 14.
That figure did not meet some analysts’ hopes of 2.0 cm growth, but for those with the condition, the drug’s potential efficacy is not at the forefront of their concerns. Rather, many individuals with achondroplasia see BioMarin attempting to solve a problem that doesn’t exist and profit off the stigmatization of their short statures.
The biotech counters that achondroplasia can lead to a host of other medical problems, such as skeletal issues that require corrective surgeries, sleep apnea, hearing loss and spinal problems.
Thursday’s news comes on the heels of the FDA handing BioMarin a surprising setback earlier this week when the agency issued a CRL for the biotech’s hemophilia A gene therapy, valrox. The program was widely regarded as a shoe-in for approval, but regulators ended up asking for 2 years of follow-on data from their Phase III trial, rather than the latest update from BioMarin’s investigators. — Max Gelman
Cold chain logistics supplier inks €49M buyout deal
As the cell and gene therapy space grows, as does a key service provider specializing in moving the temperature-sensitive material around the world.
Brentwood, TN-based Cryoport is snapping up Paris’ CRYOPDP in an all-cash deal valued at around €49 million ($57 million) — just €7 million more than the revenue CRYOPDP in 2019.
“CRYOPDP is especially strong in EMEA and APAC, where it enjoys a strong reputation as a valued specialist logistics provider to CROs (clinical research organizations), the pharmaceutical industry, and the life sciences clinical research community,” Cryoport CEO Jerrell Shelton said in a statement. “Over the coming months we anticipate focusing more attention on the fast-growing cell and gene therapy industry, where CRYOPDP will play an increasing role.”
The French company brings over 220 employees across 22 facilities in 12 countries, who collectively boast of completing 300,000 temperature-controlled shipments annually. — Amber Tong