Expected Submission of Marketing Authorization Application in First Half of 2020
LOS ANGELES, June 19, 2019 /PRNewswire/ -- CytRx Corporation (CYTR), a biopharmaceutical research and development company specializing principally in oncology, today highlighted that arimoclomol licensee Orphazyme A/S (ORPH.CO) has updated the anticipated timing for submission of its Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) seeking approval for arimoclomol in Niemann-Pick disease Type C (NPC). Based on advice received from the EMA's Scientific Advice Working Group, Orphazyme expects to submit the arimoclomol MAA in the first half of 2020.
Additional NPC data, including 12-month open-label extension study data, will become available during the second half of 2019 and must be included in the MAA submission. Orphazyme also plans to meet with the U.S. Food and Drug Administration (FDA) to discuss the pathway forward for arimoclomol in NPC this summer and the Company expects to provide an update after it has received written comments from the FDA.
"We are pleased with the progress Orphazyme is making with arimoclomol," said Eric Curtis, CytRx's President and Chief Operating Officer. "NPC is a severely debilitating and fatal disease that predominantly affects children and we commend Orphazyme for their commitment to bringing arimoclomol to patients suffering from this devastating disease."
CytRx is eligible to receive $6 million upon approval of arimoclomol in the U.S. and $4 million upon approval in Europe in Orphazyme's first non-ALS indication, plus royalties on net sales in both territories.
About Niemann-Pick Disease Type C
Niemann-Pick disease Type C (NPC) is a rare, genetic and progressive disease that impairs the ability of the body to move cholesterol and other fatty substances (lipids) inside the cells. The result is an accumulation of lipids within the body's tissue, including the brain tissue, causing damage to the affected areas. The symptoms upon onset of NPC vary from fatality during the first months after birth to a progressive disorder not diagnosed until adulthood. The disease affects neurologic and psychiatric functions as well as various internal organs. Systemic symptoms of NPC are more common in infancy or childhood and the rate of progression is usually much slower in individuals with onset of symptoms during adulthood. NPC is usually fatal and the majority of individuals with the disease die before the age of 20. NPC has been granted Orphan Drug Designation (EU and U.S.) for the treatment of NPC.
It is conservatively estimated that the number of potential NPC patients in the United States and in the EU is between 1,000 and 2,000 individuals in total. Diagnostic challenges may affect the number of potential patients. However, a treatment option could also increase awareness of the disease and assist in identifying more cases.
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, crosses the blood brain barrier, and has been studied in seven Phase 1 and three Phase 2 clinical trials. Arimoclomol is in clinical development at Orphazyme for the treatment of Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and amyotrophic lateral sclerosis.
About CytRx Corporation
CytRx Corporation (CYTR) is a biopharmaceutical company with expertise in discovering and developing new therapeutics principally to treat patients with cancer. CytRx's most advanced drug conjugate, aldoxorubicin, is an improved version of the widely used anti-cancer drug doxorubicin and has been out-licensed to NantCell, Inc. In addition, CytRx's other drug candidate, arimoclomol has been out-licensed to Orphazyme A/S (Nasdaq Copenhagen exchange: ORPHA). Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM). CytRx Corporation's website is www.cytrx.com.
About Orphazyme A/S
Orphazyme is a biopharmaceutical company focused on bringing novel treatments to patients living with life-threatening or debilitating rare diseases. Our research focuses on developing therapies for diseases caused by misfolding of proteins and lysosomal dysfunction. Arimoclomol, the company's lead candidate, is in clinical development for four orphan diseases: Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and Amyotrophic Lateral Sclerosis. The Denmark-based company is listed on Nasdaq Copenhagen (ORPHA.CO). For more information, please visit www.orphazyme.com.
This press release contains forward-looking statements. Such 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 risks and uncertainties relating to the ability of Orphazyme A/S to obtain regulatory approval for, manufacture and commercialize its products and therapies that use arimoclomol; the results of future clinical trials involving arimoclomol; the amount, if any, of future milestone and royalty payments that we may receive from Orphazyme A/S; and other risks and uncertainties described in the most recent annual and quarterly reports filed by CytRx with the Securities and Exchange Commission and current reports filed since the date of CytRx's most recent annual report. All forward-looking statements are based upon information available to CytRx on the date the statements are first published. CytRx undertakes no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
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