CytRx Notes FDA Orphazyme Regulatory Update on Arimoclomol for Niemann-Pick Disease Type C


LOS ANGELES–(COMMERCIAL THREAD) – CytRx Corporation (OTCQB: CYTR) (“CytRx”), a specialty biopharmaceutical company focused on research and development for the oncology and neurodegenerative disease categories, today noted that Orphazyme A / S (NASDAQ: ORPH ) (“Orphazyme”) announced that it has received a Complete Response Letter (“CRL”) from the United States Food and Drug Administration (“FDA”) following its review of the new drug application for the arimoclomol, a heat shock protein enhancer for the treatment of Niemann-Pick disease type C (“NPC”).

Orphazyme’s announcement revealed that the FDA issued CSF based on the need for additional qualitative and quantitative evidence to further support the validity and interpretation of the 5-Domain NPC Clinical Severity Scale (“NPCCSS” ) and, in particular, the area of ​​swallowing. Additionally, the FDA noted in the CSF that more data is needed to strengthen the confirmatory evidence beyond the single Phase 2/3 clinical trial to support the benefit and risk assessment of NDA.

A primary endpoint of the Phase 2/3 clinical trial was progression in disease severity as measured by the 5-domain NPCCSS. It is a disease-specific measure of disease progression comprising the five areas of most clinical relevance to patients with CPN, caregivers, and physicians.

Christophe Bourdon, CEO of Orphazyme declared: “We are disheartened by the outcome of the FDA review, given the urgent need for a new treatment option for NPC, but remain committed to working with regulators, with the goal of providing arimoclomol to families. who manage this difficult disease. We will focus our efforts on further European regulatory approval, with a CHMP opinion expected in Q4 2021 and possible marketing authorization in Q1 2022. We are evaluating the way forward in the US in partnership with the FDA. In the short term, we will need to significantly reduce our costs and freeze all company efforts unrelated to clinical and regulatory activities to support NPC approval. ”

Orphazyme revealed that, as noted in its 2020 Annual Report, the initial outlook for the year was subject to various risks and uncertainties, including, but not limited to, the timing of regulatory decisions, the success of commercial efforts and Orphazyme development activities. The outcome of the FDA decision has a significant influence on Orphazyme’s outlook for the year 2021. Orphazyme’s cash position at the end of 2021 is now expected to be around $ 8 million (previously $ 56 million). millions of dollars).

Orphazyme has indicated that it will provide an update and more information in the coming weeks.

About CytRx Corporation

CytRx Corporation (OTCQB: CYTR) is a biopharmaceutical company specializing in the discovery and development of new therapies primarily to treat patients with cancer and neurodegenerative diseases. CytRx’s drug candidate, arimoclomol, was sold to Orphazyme A / S (Nasdaq Copenhagen Exchange: ORPHA.CO) in exchange for milestone payments and royalties. Orphazyme develops arimoclomol in Niemann-Pick disease type C (“NPC”) and Gaucher disease. Learn more at

About Orphazyme

Orphazyme is a biopharmaceutical company focused on providing new treatments for patients living with rare, life-threatening or debilitating diseases. Their research is focused on developing therapies for diseases caused by protein misfolding, including lysosomal overload diseases. Arimoclomol, the Company’s lead candidate, is in clinical development in Niemann-Pick disease type C and Gaucher disease. Orphazyme is headquartered in Denmark and operates in the United States and Switzerland. Orphazyme shares are listed on NASDAQ: ORPH. For more information, please visit

About Niemann-Pick disease type C

Niemann-Pick type C disease (NPC) is a rare, genetic, progressively debilitating and often fatal neurovisceral disease. It belongs to a family known as lysosomal overload diseases and is caused by mutations leading to a defective NPC protein. As a result, the lipids that are normally eliminated by the lysosome accumulate in tissues and organs, including the brain, and lead to the pathology of the disease. We estimate the incidence of NPC at one live birth in 100,000 and the number of NPC patients in the United States and Europe at approximately 1,800 people. There are no approved treatments for NPC in the United States

About arimoclomol

Arimoclomol is an investigational drug candidate that enhances the production of heat shock proteins (HSPs). HSPs can rescue defective misfolded proteins, remove protein aggregates, and improve lysosome function. Arimoclomol is administered orally, crosses the blood brain barrier, and has been studied in seven phase 1, four phase 2, and one pivotal phase 2/3 clinical trials. Arimoclomol is in clinical development at Orphazyme for the treatment of NPCs and Gaucher disease. Arimoclomol has received orphan drug designation for NPC in the United States and the EU, as well as expedited designation from the US Food and Drug Administration (FDA) for NPC. In addition, arimoclomol has received breakthrough therapy designation and rare pediatric disease designation from the FDA for NPC.

Forward-looking statements

This press release contains forward-looking statements, including statements relating to the potential obtaining of EMA and FDA approval for arimoclomol, the potential receipt by the Company of future milestone and royalty payments. Orphazyme and the realization of long-term value for the shareholders of the Company. These statements involve risks and uncertainties that could cause actual events or results to differ materially from the events or results described in the forward-looking statements, including the risks and uncertainties relating to Orphazyme’s ability to achieve the regulatory approval for, manufacture and market its products. and therapies that use arimoclomol; results of clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments we may receive from Orphazyme; and other risks and uncertainties described in the most recent annual and quarterly reports filed by the Company with the SEC and current reports filed since the date of the most recent annual report of the Company. All forward-looking statements are based on information available to the Company at the date of their first publication. The Company assumes no obligation to publicly update or revise forward-looking statements, whether as a result of new information, future events or otherwise.

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