RBC sees nod for Amylyx's ALS drug as a sign of FDA flexibility

Design Therapeutics

30/09/2022 18:04

** Brokerage RBC Capital says U.S. health regulator's nod
for Amylyx's  AMLX.O  ALS drug is an "important litmus test" for
continued regulatory flexibility in rare disease indications
 urn:newsml:reuters.com:*:nL4N3103XX 
        ** RBC sees favorable readthroughs such as from ALS
drugmakers in the regulatory process like Biogen  BIIB.O /Ionis
 IONS.O  and Acadia  ACAD.O  to allow for continued regulatory
permissiveness 
    ** Says FDA's full approval for AMLX signals a single study
can be sufficient if it shows statiscally significant benefits -
brokerage
    ** RBC believes AMLX approval being based on a 135 patient
mid-stage study could enable cos like Edgewise  EWTX.O  and
Design  DSGN.O  to explore similar study designs for their ALS
drug candidates 
    ** Says inclusion of follow-up analyses and statistical
methodology in AMLX's drug label, gives ALS drugmakers like
Sarepta  SRPT.O  and BIIB/IONS an opportunity to make a
compelling argument for approval 
    ** Brokerage notes FDA's flexibility might be highest in ALS
due to lack of options and disease severity ; may not extend
across all rare neuro indications
    ** All 5 brokerages covering stock rate it "hold" or higher;
their median PT is $46
    ** As of last close, AMLX up ~67% since market debut on
Nasdaq in January
 (Reporting by Bhanvi Satija in Bengaluru)
 ((Bhanvi.Satija@thomsonreuters.com; Outside U.S. +91
9873062788;))