MIPLYFFA is the first FDA-approved treatment for Niemann-Pick disease type C (NPC), an ultra-rare and progressive neurodegenerative disease MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adults and pediatric patients 2 years of age and older Zevra receives rare pediatric disease priority review voucher in conjunction with approval Company launches AmplifyAssist™ patient support program Conference call and webcast set for 8:00 a.m. EDT o ...

New clinical efficacy and safety data for arimoclomol as a possible treatment for Niemann-Pick disease type C, including from long-term and real-world settings, demonstrate clinically meaningful reduction in disease progression Arimoclomol was well tolerated during the Open Label Extension trial and Early Access Program with no safety signals identified PK modeling studies showed that administration of OLPRUVA while fasting vs fed results in higher drug exposure which may allow for lower effective dosages w ...

CELEBRATION, Fla., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that Neil F. McFarlane, President and Chief Executive Office of Zevra, will present at upcoming investor conferences during the month of September 2024. Additionally, management will be available for one-on-one meetings with registered attendees at each conference. H.C. Wainwright 26th Annual Global Investment Conference in New York, NY, ...

CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably (11 yes, 5 no) that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC). "We are extremely pleased with the committee's recognition of the benefits of arimo ...

CELEBRATION, Fla., July 30, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and audio webcast on Tuesday, August 13, 2024, at 4:30 p.m. ET, to review its corporate and financial results for the second quarter of 2024. The link to the audio webcast will be accessible via the Investor Relations section of the Company's website, https://investors.zevra.com/. To join the meeting by ...

CELEBRATION, Fla., July 30, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced that it will host a conference call and audio webcast on Tuesday, August 13, 2024, at 4:30 p.m. ET, to review its corporate and financial results for the second quarter of 2024. The link to the audio webcast will be accessible via the Investor Relations section of the Company's website, https://investors.zevra.com/. To join the meeting by ...

Zevra Therapeutics, Inc. (ZVRA) announced that the FDA has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee ("GeMDAC") to review the company's new drug application (NDA) for pipeline candidate arimoclomol. NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability of the body to transport cholesterol and other lipids within the cell, leading to an accumulation of these substances in various ...

The stock rallied after the FDA indicated that it would convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee to review the company's new drug application (NDA) for pipeline candidate arimoclomol. The NDA is seeking approval for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C. The stock currently carries a Zacks Rank #3 (Hold). You can see the complete list of today's Zacks Rank #1 (Strong Buy) stocks here >>>> While earnings an ...

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period From to Commission File Number: 001-36913 Zevra Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Juris ...

Zevra Therapeutics, Inc. (NASDAQ:ZVRA) Q1 2024 Earnings Conference Call May 8, 2024 8:00 AM ET Company Participants Nichol Ochsner - VP, Investor Relations & Corporate Communications Neil Mcfarlane - President, CEO & Director LaDuane Clifton - CFO, Treasurer, Secretary Josh Schafer - Chief Commercial Officer & EVP, Business Development Adrian Quartel - Chief Medical Officer Conference Call Participants Tim Lugo - William Blair & Company Jonathan Aschoff - ROTH MKM Louise Chen - Cantor Fitzgerald Oren Livnat ...