CELEBRATION, Fla., Aug. 02, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NASDAQ: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 arimoclo ...

文章核心观点 - 美国食品药品监督管理局(FDA)Genetic Metabolic Diseases Advisory Committee(GeMDAC)投票支持arimoclomol治疗尼曼-皮克病C型(NPC)的有效性[1][2][3] - 公司对arimoclomol获批持乐观态度,认为临床数据支持其治疗NPC的疗效[2][3] - arimoclomol已获FDA授予孤儿药、快速通道、突破性疗法和罕见儿童疾病指定,欧洲药品管理局也授予其治疗NPC的孤儿药品地位[5] NPC疾病概述 - NPC是一种罕见、进行性和神经退行性的溶酶体贮积障碍性疾病,由NPC1或NPC2基因突变导致[4] - 患者会因身体和认知功能受限而失去独立生活能力,出现言语、认知、吞咽、行走和精细运动技能障碍[4] - 疾病进程不可逆转,可在几个月内致命,也可能需要多年才被诊断并逐步恶化[4] 公司概况 - 公司是一家专注于罕见疾病的生物制药公司,致力于利用科学、数据和患者需求开发创新疗法[6] - 公司采取独特的数据驱动开发和商业化策略,克服复杂的药物开发挑战,为罕见疾病患者带来新的治疗方案[6]

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.The NDA is seeking approval for arimoclomol, an orally delivered, first-in-class treatment for Niemann-Pick disease type C (“NPC”).NPC is an ultra-rare, progressive, and neurodegenerative lysosomal storage disorder characterized by an inability ...

Zevra Therapeutics (ZVRA) shares rallied 21.9% in the last trading session to close at $5.48. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 3.2% loss over the past four weeks. 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. Th ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 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 ...

Exhibit 99.1 Zevra Therapeutics Reports First Quarter 2024 Financial Results and Corporate Updates Full commercial launch of OLPRUVA at the end of January 2024, with market access growing to ~75% of covered lives as of May 1, 2024 Arimoclomol NDA review underway with PDUFA date of September 21, 2024 Reported positive topline results from KP1077 Phase 2 Trial in idiopathic hypersomnia, with full data to be presented at SLEEP 2024 in early June 2024 Conference call scheduled for today, May 8, 2024, at 8:00 a. ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ____ to _____ Commission File No. 001-36913 Zevra Therapeutics, Inc. (Exact Name of Registrant as Specified in Its Charter) Delaware 20-5894398 (State or Other Jurisdiction of I ...

Exhibit 99.1 Zevra Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Updates Launch of OLPRUVA® underway Topline results from KP1077 phase 2 study demonstrate clinically meaningful benefits for key IH symptoms Q4 2023 net revenue of $13.2M, and FY 2023 net revenue of $27.5M Conference call scheduled for today, March 28, 2024, at 4:30 p.m. ET Celebration, FL – March 28, 2024 – Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics c ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2023 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 J ...