Orphazyme provides regulatory update following Type A meeting with FDA on arimoclomol in Niemann-Pick disease type C
01 November 2021 - 7:00AM
Orphazyme provides regulatory update following Type A meeting with
FDA on arimoclomol in Niemann-Pick disease type C
Orphazyme A/SCompany
announcement No.
24/2021Inside informationCompany Registration No. 32266355
-
Progress made in understanding potential resolution of topics
outlined in Complete Response Letter, including need for additional
data to support NDA
-
FDA recommends the company provides supplemental information and
analyses, and the FDA offers to have further interactions to
identify a path to resubmission for arimoclomol in NPC
Copenhagen –
October 31,
2021 – Orphazyme
A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company, today
provides an update on the regulatory status in the United States of
its investigational product arimoclomol for Niemann-Pick disease
type C (NPC) following a recently held Type A meeting with the U.S.
Food and Drug Administration (FDA).
The Company had a collaborative dialogue with
the FDA during the Type A meeting, which was held in order to
discuss the key topics in the Complete Response Letter (CRL) issued
by the FDA in June 2021. The CRL was issued based on the need for
additional confirmatory evidence as well as additional qualitative
and quantitative evidence to further substantiate the validity of
the 5-domain NPC Clinical Severity Scale (NPCCSS), in particular
the swallow domain, in the context of a lack of significant
findings when using the FDA’s preferred and recommended statistical
approach.
The Type A meeting resulted in the following
take-aways:
- The FDA recommended
that the Company submit additional data, information, and analyses
to address certain topics in the CRL and engage in further
interactions with the FDA to identify a pathway to
resubmission.
- The FDA concurred
with the Company’s proposal to remove the cognition domain from the
NPCCSS endpoint, with the result that the primary endpoint is
permitted to be recalculated using the 4-domain NPCCSS, subject to
the submission of additional requested information which the
Company intends to provide. To bolster the confirmatory evidence
already submitted, the FDA affirmed that it would require
additional in vivo or pharmacodynamic (PD)/pharmacokinetic (PK)
data; the Company is considering the optimal path forward to
address the FDA’s requests.
“We are pleased to have gained a greater
understanding from the FDA on the information that could address
topics in the CRL. This is good progress. We are encouraged by the
FDA’s request to submit additional information and the invitation
to further engage to discuss our approach and potential path
forward. While we have not yet established a path to resubmission,
our team will now work on putting a plan in place to discuss with
the FDA during our next interactions and we will share more details
about our strategy as and when appropriate” said Christophe
Bourdon, Chief Executive Officer, Orphazyme A/S. He added, “We
firmly believe in the establishment of a positive benefit-risk
balance for arimoclomol and will continue to support the NPC
community and our early access programs.”
The EU Marketing Authorisation Application (MAA)
for arimoclomol in NPC was filed with the European Medicines Agency
in November 2020. As previously communicated, an opinion from the
Committee for Medicinal Products for Human Use (CHMP) on this
application is expected in Q1 2022.
For additional information, please
contact:
Orphazyme A/S
Anders Vadsholt, Chief Financial
Officer +45 2898
9055
About Niemann-Pick disease type
CNiemann-Pick disease type C (NPC) is a rare, genetic,
progressively debilitating, and often fatal neurodegenerative
disease. It belongs to a family known as lysosomal storage
disorders and is caused by mutations leading to defective NPC
protein. As a consequence, lipids that are normally cleared by the
lysosome accumulate in tissues and organs, including the brain, and
drive the disease pathology. We estimate the incidence of NPC to be
one in 100,000 live births and the number of NPC patients in the
United States and in Europe to be approximately 1,800 individuals.
There are no approved treatments for NPC in the U.S.
About arimoclomol Arimoclomol is
an investigational drug candidate that amplifies the production of
heat shock proteins (HSPs). HSPs can rescue defective misfolded
proteins, clear protein aggregates, and improve the function of
lysosomes. Arimoclomol is administered orally, and has now been
studied in 10 Phase 1, four Phase 2, and three pivotal Phase 2/3
trials. Arimoclomol has received Orphan Drug Designation (ODD) for
NPC in the US and EU. Arimoclomol has received Fast-Track
Designation (FTD), Breakthrough Therapy Designation (BTD), and Rare
Pediatric Disease Designation (RPDD) from the U.S. Food and Drug
Administration (FDA) for NPC. On June 17, 2021, Orphazyme received
a Complete Response Letter from the FDA regarding its New Drug
Application for arimoclomol for the treatment of NPC. A marketing
authorization application (MAA) for arimoclomol in NPC has been
filed with the European Medicines Agency and is under review.
About Orphazyme
A/S Orphazyme is a late-stage biopharmaceutical
company developing arimoclomol for Niemann-Pick disease type C
(NPC). Orphazyme is headquartered in Denmark and has operations in
the U.S. and Switzerland. ADSs representing Orphazyme’s shares are
listed on Nasdaq U.S. (ORPH) and its shares are listed on Nasdaq
Copenhagen (ORPHA).
Forward-looking
statement This company announcement may contain
certain forward-looking statements under the U.S. Private
Securities Litigation Reform Act of 1995 and otherwise, including
the next steps required to obtain regulatory approval for
arimoclomol in the United States following the FDA Type A meeting
and the length or time and cost required therefor, and the timing
of the opinion from the CHMP. Although the Company believes its
expectations are based on reasonable assumptions, all statements
other than statements of historical fact included in this company
announcement about future events are subject to (i) change without
notice and (ii) factors beyond the Company’s control. These
statements may include, without limitation, any statements preceded
by, followed by, or including words such as “target,” “believe,”
“expect,” “aim,” “intend,” “may,” “anticipate,” “estimate,” “plan,”
“project,” “will,” “can have,” “likely,” “should,” “would,”
“could”, and other words and terms of similar meaning or the
negative thereof. Forward-looking statements are subject to
inherent risks and uncertainties beyond the Company’s control that
could cause the Company’s actual results, performance, or
achievements to be materially different from the expected results,
performance, or achievements expressed or implied by such
forward-looking statements, including the risks and uncertainties
that are described in the Risk Factors section of the Company’s
Annual Report on Form 20-F for the year ended December 31, 2020
filed with the U.S. Securities and Exchange Commission (SEC) on
March 2, 2021, the Company’s Report on Form 6-K filed with the SEC
on June 11, 2021, and other filings Orphazyme makes with the SEC
from time to time. These documents are available on the “Investors
& Media” section of Orphazyme’s website at www.orphazyme.com.
Except as required by law, the Company assumes no obligation to
update these forward-looking statements publicly, or to update the
reasons actual results could differ materially from those
anticipated in the forward-looking statements, even if new
information becomes available in the future.
- 24-2021 Orphazyme provides regulatory update following Type A
meeting with FDA on arimoclomol in NPC
Orphazyme A/s (LSE:0CUM)
Historical Stock Chart
From Mar 2024 to Apr 2024
Orphazyme A/s (LSE:0CUM)
Historical Stock Chart
From Apr 2023 to Apr 2024