Feb. 14 at 12:27 AM
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Biohaven CEO calls recent FDA decisions 'a systemic problem'
'It's really a dire time for patients': Biohaven CEO says FDA red tape is blocking access to rare disease treatments
After a string of high-profile regulatory rebukes, Biohaven’s CEO believes red tape is getting in the way of patient well-being, particularly for those with rare diseases.
Months after the FDA rejected Biohaven’s bid to approve its spinocerebellar ataxia (SCA) therapy, CEO Vlad Coric, M.D., says the decision is part of a broader shift at the agency that may stifle innovation.
“This is a systemic problem that we saw at the FDA last year and continue to see,” Coric told Fierce Biotech on Wednesday.
“This is not a Biohaven issue,” he continued, citing “other rare disease issues,” such as the FDA’s refusal to approve Regenxbio’s Hunter syndrome gene therapy based on concerns about several trial design features, such as the use of a natural history control arm.
Other issues include Stealth BioTherapeutics’ laborious back-and-forth with the regulator that ultimately ended in an approval for the ultrarare Barth syndrome after numerous rejections. The tides eventually changed for StealthBio after the FDA faced wide-ranging public outcry regarding its delays and prior snubs for the rare disease candidate.
Biohaven’s CEO also mentioned the FDA’s hotly contested decision to refuse review for Moderna's next-gen flu vaccine this week.
“It’s really a dire time for patients,” he said, explaining that Biohaven is currently appealing the agency’s decision. But, if the FDA doesn’t provide a path forward, the program will cease to exist, he said.
more:
https://www.fiercebiotech.com/biotech/its-really-dire-time-patients-biohaven-ceo-says-fda-red-tape-blocking-access-rare-disease
