Spirovant's SPIRO-2101 gains special designations from FDA

25 September 2020
dainippon-big

Gene therapy specialist Spirovant Sciences, which is ultimately owned by Japan’s Sumitomo Dainippon Pharma (TYO: 4506), says that the US Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease designations to its lead product candidate, SPIRO-2101, for the treatment of cystic fibrosis.

SPIRO-2101, an inhaled adeno-associated virus (AAV) gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator. SPIRO-2101 contains an evolved AAV capsid engineered to have high tropism to human airway epithelia. This treatment is designed for these patients who currently have no approved modulator therapies.

"Cystic fibrosis is an incredibly debilitating and devastating disease," said Joan Lau, chief executive of Spirovant Sciences, adding: "Receiving Orphan Drug and Rare Pediatric Disease Designations for SPIRO-2101 underscores the urgency for a treatment option for these patients, particularly those with very rare and more severe types of cystic fibrosis, the nonsense mutation subtype. With these important regulatory milestones, combined with our amazing and growing team, we are well positioned to advance SPIRO-2101 into the clinic and offer hope to these patients."

This article is accessible to registered users, to continue reading please register for free.  A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Login to your account

Become a subscriber

 

£820

Or £77 per month

Subscribe Now
  • Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
  • Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
  • Daily roundup of key events in pharma and biotech.
  • Monthly in-depth briefings on Boardroom appointments and M&A news.
  • Choose from a cost-effective annual package or a flexible monthly subscription
The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK



Today's issue

Company Spotlight





More Features in Biotechnology