ORPHAZYME RECEIVES ORPHAN DRUG DESIGNATION TO ARIMOCLOMOL FOR INCLUSION BODY MYOSITIS FROM THE U.S. FDA
(Thomson Reuters ONE) -
Orphazyme A/S
Ole Maaløes Vej 3
DK-2200 Copenhagen N
www.orphazyme.com
Company Registration No. 32266355
7 November 2017
Orphazyme A/S ("Orphazyme" or the "Company"), a Danish biotech company with a
late stage orphan drug pipeline, today announced that the U.S. Food and Drug
Administration ("FDA"), Office of Orphan Products Development, has granted an
orphan drug designation to arimoclomol for the treatment of the orphan disease
Inclusion Body Myositis ("IBM"). As described in the offering circular published
by Orphazyme A/S on 6 November 2017, the Company applied to the FDA for the
designation in September 2017.
The Company focuses, among others, on sporadic inclusion body myositis ("sIBM")
and has already obtained an EU orphan drug designation for sIBM from the
European Medicines Agency ("EMA"), which was granted in 2016.
About U.S. orphan drug designation
The United States Congress passed the Orphan Drug Act in 1983 to provide
incentives for investment in treatments for rare conditions. Companies that
receive orphan drug designation are entitled to several advantages, including
the possibility of free of charge advice from the FDA and certain financial
benefits, such as R&D tax credits (which is not part of the company's current
plans) and exemptions or reductions in regulatory submission fees. If a drug
candidate with orphan drug designation is approved by the FDA upon completion of
clinical trials, it may receive orphan drug status providing the orphan drug
with market exclusivity for seven years in the United States.
About sIBM
The disease is one of several neuromuscular diseases and is one of the most
common wasting muscle disorders, affecting patients in the later stages of life,
typically over the age of 50 years. The size of the patient population in Europe
and the United States is not fully elucidated but has been conservatively
estimated to be between 7,000 and 15,000 individuals. No effective treatment is
currently available.
For additional information, please contact:
Anders Hinsby, CEO +45 31 44 31 39
About Orphazyme A/S
Orphazyme is a Danish biotech company with a late stage orphan drug pipeline,
developing new treatment options for orphan protein misfolding diseases. The
Company was founded in 2009 based on early scientific discovery in heat shock
proteins ("HSPs"). Since inception, the Company has translated scientific
discovery into a late stage clinical development programme. The Company is
headquartered in Copenhagen and currently has 30 employees.
The lead candidate arimoclomol is in development as a potential treatment for
four orphan diseases; two neuromuscular diseases, sporadic Inclusion Body
Myositis ("sIBM") and Amyotrophic Lateral Sclerosis ("ALS"), and two lysosomal
storage diseases, Niemann Pick type C ("NPC") and Gaucher disease.
Press release (PDF):
http://hugin.info/174047/R/2147817/823769.pdf
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Orphazyme A/S via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 07.11.2017 - 15:12 Uhr
Sprache: Deutsch
News-ID 567000
Anzahl Zeichen: 4162
contact information:
Town:
Copenhagen N
Kategorie:
Business News
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