Orphazyme fails to win U.S. backing for arimoclomol drug (updated)

Strategic Partners A/S

18/06/2021 07:05

(Recasts, adds background, detail)
    June 18 (Reuters) - Danish pharmaceutical company Orphazyme
 ORPHA.CO  has failed to win support from the U.S. Food and Drug
Administration (FDA) for its arimoclomol drug, a treatment
designed for the Niemann-Pick disease type C genetic disorder,
the company said on Friday.
    Following the FDA's review, Orphazyme predicted its revenue
for the year will be lower than previously expected and the
operating loss significantly wider, forcing the company to cut
costs.
    Orphazyme said the FDA in a letter had said additional
qualitative and quantitative evidence was needed for the drug's
effectiveness.
    Orphazyme, which is dually listed in Copenhagen and New
York, now expects an operating loss of 670 million crowns to 700
million crowns ($107 million-$112 million) in 2021, against a
previous forecast for a loss of 100 million-150 million crowns.
    "As representative for Orphazyme's shareholders and as a
shareholder myself, I am extremely disappointed," deputy board
chairman Bo Jesper Hansen said in a statement.
    The drugmaker will continue to seek approval for the
treatment in Europe, it said.
    The company's share price has risen threefold this month,
earnings a reputation as a "meme stock" during frenzied trading
as many smaller investors positioned themselves for the outcome
of the FDA's decision.  urn:newsml:reuters.com:*:nL5N2NZ5J4 urn:newsml:reuters.com:*:nL5N2NW4IH 
    ($1 = 6.2456 Danish crowns)

 (Reporting by Boleslaw Lasocki in Gdansk and Terje Solsvik in
Oslo, Editing by Sherry Jacob-Phillips and Jason Neely)
 ((gdansk.newsroom@thomsonreuters.com; +48 58 772 0920;))