Brief: Orphazyme Provides Regulatory Update From FDA On Arimoclomol For Niemann-Pick Disease Type C

Strategic Partners A/S

18/06/2021 06:07

June 18 (Reuters) - Orphazyme A/S  ORPHA.CO :
    * REG-ORPHAZYME PROVIDES REGULATORY UPDATE FROM FDA ON
ARIMOCLOMOL FOR NIEMANN-PICK DISEASE TYPE C
    * HAS RECEIVED A COMPLETE RESPONSE LETTER (CRL) FROM U.S.
FOOD AND
DRUG ADMINISTRATION (FDA)
    * CHANGED OUTLOOK FOR 2021
    * OUTCOME OF FDA DECISION HAS SIGNIFICANT INFLUENCE ON
ORPHAZYME'S
OUTLOOK FOR FULL-YEAR 2021.
    * NOW EXPECTS OPERATING EXPENSES OF DKK 700-720 MILLION
(PREVIOUSLY DKK 800-850 MILLION) RESULTING IN AN EXPECTED
OPERATING LOSS OF DKK 670-700 MILLION (PREVIOUSLY DKK 100-150
MILLION)
    * FDA NOTED IN CRL THAT ADDITIONAL DATA ARE NEEDED TO
BOLSTER
CONFIRMATORY EVIDENCE BEYOND SINGLE PHASE 2/3 CLINICAL TRIAL
    * CASH POSITION AT YEAR-END 2021 IS EXPECTED TO BE APPROX.
DKK 50
MILLION (PREVIOUSLY DKK 350 MILLION).
    * PRIMARY ENDPOINT OF PHASE 2/3 CLINICAL TRIAL WAS
PROGRESSION IN
DISEASE SEVERITY AS MEASURED BY 5-DOMAIN NPCCSS.
    * WE WILL FOCUS OUR EFFORTS ON PURSUING EUROPEAN REGULATORY
APPROVAL, WITH CHMP OPINION EXPECTED IN Q4 2021 AND POTENTIAL
MARKETING AUTHORIZATION IN Q1 2022
    * IN SHORT-TERM, WE WILL NEED TO REDUCE OUR COSTS
SUBSTANTIALLY
AND FREEZE ALL COMPANY EFFORTS NOT RELATED TO CLINICAL AND
REGULATORY ACTIVITIES TO SUPPORT APPROVAL FOR NPC
    * WE ARE ASSESSING POTENTIAL PATH FORWARD IN U.S. IN
PARTNERSHIP
WITH FDA.
    * IN SHORT-TERM, WILL NEED TO REDUCE COSTS SUBSTANTIALLY AND
FREEZE ALL COMPANY EFFORTS NOT RELATED TO CLINICAL AND
REGULATORY ACTIVITIES
    * AS REPRESENTATIVE FOR ORPHAZYME'S SHAREHOLDERS AND AS A
SHAREHOLDER MYSELF, I AM EXTREMELY DISAPPOINTED - DEPUTY
CHAIRMAN

Source text for Eikon:  ID:nGNE4bcTwg 
Further company coverage:  ORPHA.CO 

 (Gdansk Newsroom)
 ((gdansk.newsroom@thomsonreuters.com; +48 58 769 66 00;))