Zevra Therapeutics, Inc. (NASDAQ: ZVRA), slumped Friday. The company, a commercial-stage concern focused on bringing life-changing therapeutics to people living with rare diseases, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a negative opinion regarding the Marketing Authorization Application (MAA) for arimoclomol for the treatment of Niemann-Pick disease type C (NPC), a rare, progressive and life-threatening neurodegenerative disorder.
Under European regulatory procedures, the Company plans to request a re-examination of the CHMP opinion and submit a detailed explanation outlining the basis for the request.
“While the review did not result in the outcome we had hoped for, we are committed to investigating appropriate pathways to bring arimoclomol to patients in Europe,” said CEO Neil F. McFarlane.
“We appreciate the important role the CHMP plays in assessing therapies across Europe, and we remain confident in the totality of evidence supporting the potential of arimoclomol to make a meaningful difference in the lives of patients living with NPC.”
The release goes onto say “Zevra remains steadfast in its commitment to the NPC community and believes that patients facing this devastating disease deserve access to potential treatment options.
“Through its global Expanded Access Program (EAP), the Company continues to support eligible patients to advance access to arimoclomol and generate additional real-world evidence of its clinical impact.”
ZVRA shares let go of $2.66, or 21.3%, to $9.86.
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