Federal Register scheduled news release on 10/15 for Issuance of Priority Review Voucher. The Food and Drug Administration (FDA) is announcing the issuance of a priority review voucher to the sponsor of a rare pediatric disease product application. The Federal Food, Drug, and Cosmetic Act (FD&C Act) authorizes FDA to award priority review vouchers to sponsors of approved rare pediatric disease product applications that meet certain criteria. FDA is required to publish notice of the award of the priority review voucher. FDA has determined that MIPLYFFA (arimoclomol), approved on September 20, 2024, manufactured by Zevra Denmark AS, meets the criteria for a priority review voucher.

Thoughts?

Also, speed run, you still holding!

https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-participate-rare-disease-panel-2024-maxim

https://finance.yahoo.com/news/investors-zevra-therapeutics-nasdaq-zvra-104303458.html

KP1077 demonstrates clinically meaningful benefits for key IH symptoms

Top-line data provide key information for the design of a Phase 3 study

Anyone have any expectations for tomorrow’s call ?

https://stocks.apple.com/AFu_R6TLuR6-r7f_Cdo5w3Q

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=214927

They announced it yesterday.

ZVRA completely halted market wide today?

Zevra 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.

ArimoclomolNiemann-Pick disease Type C (NPC)

FDA Advisory Committee Meeting:
August 2, 2024
Importance: This meeting is crucial as the advisory committee will review and provide recommendations on the drug application. Their feedback can significantly influence the FDA’s final decision.

PDUFA Date

September 21, 2024

Importance: This is the deadline by which the FDA is expected to make a decision on the drug application. The PDUFA date extension indicates that the FDA has taken additional time to review the submission, which could be due to the need for more data or other considerations.

* Heaps of insider buying past year
* This company has an excellent track record (88%+) LOA and POP historically.

There used to be weekly updated script numbers on Azstarys. Does anyone know how the numbers are doing now?

The U.S. Food and Drug Administration (FDA) has indicated that it will convene a meeting with the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) on August 2, 2024, to review the New Drug Application (NDA) for arimoclomol as an orally delivered, first-in-class treatment for Niemann-Pick disease type C (NPC).

Rahsaan W. Thompson appointed as Chief Legal Officer, Secretary and Compliance Officer

Alison Peters appointed as Chief People Officer

“The results of the Phase 2 clinical trial support the safety and tolerability of KP1077 as measured by the primary endpoint of the study,” stated Christopher Drake, PhD, FAASM, DBSM, Principal Investigator of the study. “The clinically meaningful impact for both the safety and efficacy were well demonstrated by patients showing significant improvements in IH symptom severity such as sleep inertia, excessive daytime sleepiness (EDS), and patient-reported IH-specific outcomes.”

Can someone enlighten me on the upcoming catalysts for Zevra in the coming 3-6 months?

And also why is there not a bigger focus on Azstarys sales as this was suppose to be a potential blockbuster? Are they not performing as expected?

https://investors.zevra.com/news-releases/news-release-details/zevra-therapeutics-report-first-quarter-2024-results-may-8-2024

Neil F. McFarlane, Zevra’s President and Chief Executive Officer, has issued a Letter to Stockholders providing a review of key accomplishments and outlook for 2024.