MIPLYFFA
Latest News
U.S. FDA approves MIPLYFFA™ (arimoclomol) for the Treatment of NPC
Zevra Therapeutics is pleased to share the U.S. Food and Drug Administration (FDA) has approved MIPLYFFA™, for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. MIPLYFFA™ is the first therapy indicated specifically for the treatment of NPC, and is currently the only approved treatment for this disease.
Read the letter to the community.
Read the press release.
Read the FDA press release.
09/20/2024
Update from Zevra Therapeutics
Zevra Therapeutics is pleased to share a letter to the community following Friday’s positive vote, which also includes a link to the press release.
The announcement shares that the U.S. Food and Drug Administration (FDA) Genetic Metabolic Diseases Advisory Committee (GeMDAC) voted favorably that the data support that arimoclomol is effective in the treatment of patients with Niemann-Pick disease type C (NPC).
Zevra will continue to work closely with the FDA and respond to any outstanding questions they may have in the coming weeks. The assigned Prescription Drug User Fee Act (PDUFA) action date for arimoclomol is September 21, 2024.
Read the complete press release.
Read the letter to the community.
08/05/2024
Update from Zevra Therapeutics
Zevra Therapeutics shared today that the U.S. Food and Drug Administration (FDA) announced that the recently formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) will meet on August 2, 2024 to review Zevra Therapeutics’ New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick type C (NPC). The meeting will be open to the public via livestream on YouTube.
Read the complete press release.
Read the letter to the community.
07/9/2024
Update from Zevra Therapeutics
Zevra Therapeutics has shared an FDA update on the PDUFA action date for Arimoclomol as a treatment for Niemann-Pick type C.
Read the complete announcement.
Read the FDA update.
03/04/2024
Zevra Therapeutics Receives FDA Acceptance of Resubmission of NDA for Arimoclomol as a Treatment for Niemann-Pick Disease Type C
Zevra Therapeutics has announced the U.S. Food and Drug Administration’s (FDA) acceptance of the resubmission of the New Drug Application (NDA) for arimoclomol for treatment of Niemann-Pick disease type C. Read the complete announcement and Zevra’s message to the Niemann-Pick community below.
Read the complete announcement.
Read the message to the community.
01/08/2024
Zevra Therapeutics Announces Resubmission of Arimoclomol New Drug Application to the U.S. Food And Drug Administration
Zevra Therapeutics is pleased to announce the resubmission of its New Drug Application (NDA) for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick Disease type C (NPC) to the U.S. Food and Drug Administration (FDA) on December 22, 2023. Based on standard NDA resubmission review timelines, an acknowledgment letter from the FDA that the resubmission is complete and setting the PDUFA date is expected within 30 days and anticipate a six-month review period thereafter.
Read the complete announcement and Zevra’s message to the Niemann-Pick community below.
Read the complete announcement.
Read the message.
12/27/2023
Zevra Announces Completion of Acer Therapeutics Acquisition
Zevra announces the completion of its acquisition of Acer Therapeutics. Zevra states they are committed to the arimoclomol expanded access program as a much-needed treatment of NPC, and their goal is to resubmit the arimoclomol New Drug Application (NDA) to the FDA by the end of 2023. Read the complete announcement.
Please contact Joslyn Crowe, NNPDF Executive Director, with any questions.
11/20/2023
Zevra Therapeutics Celebrates and Supports Global Niemann-Pick Disease Awareness Day on October 19th and Niemann-Pick Disease Awareness Month Throughout October
Zevra Therapeutics, Inc. proudly supports the Niemann-Pick community on Global Niemann-Pick Disease Awareness Day and throughout Niemann-Pick Disease Awareness Month. Patient organizations like the International Niemann-Pick Disease Alliance (INPDA) and the National Niemann-Pick Disease Foundation (NNPDF) provide essential support for people living with Niemann-Pick disease type C (NPC) and their families and advocate for needed treatments. Read the complete announcement.
10/19/2023
Zevra Therapeutics Appoints Neil F. McFarlane as President, Chief Executive Officer and Director
Zevra Therapeutics has announced that Neil F. McFarlane has been named President and Chief Executive Officer of Zevra and appointed to the Board of Directors. Read the complete announcement.
Zevra has also shared a letter to the NPC Community. Read the letter.
10/11/2023
Zevra Therapeutics Statement to the Community
Zevra Therapeutics has shared the following statement to the community with the NNPDF announcing their intention to acquire Acer Therapeutics. Both companies are focused on the development and commercialization of therapies for rare diseases with significant unmet medical needs. Zevra remains steadfast to their key priorities to the NPC community. Read the complete statement.
Additionally, the press release can be found here.
08/23/2023
Zevra Therapeutics to Acquire Acer Therapeutics, Expanding its Rare Disease Portfolio and Adding Commercial Product
Zevra Therapeutics, Inc. and Acer Therapeutics Inc. today announced the companies have entered into a definitive agreement pursuant to which Zevra would acquire Acer in a merger transaction.
08/23/2023
Zevra Therapeutics to Participate in the 2023 NNPDF Family Support & Medical Conference
Zevra Therapeutics announced their support of the upcoming National Niemann-Pick Disease Foundation’s (NNPDF) Family Support and Medical Conference. Members of the Zevra team will attend this event which is being held in Orlando, Florida, from July 20 through July 22, 2023. Read the press release.
07/19/2023
KemPharm Announces Corporate Name Change to Zevra Therapeutics
KemPharm has shared the following press release with the NNPDF announcing its name change to Zevra Therapeutics. Read press release.
Zevra Therapeutics (formerly KemPharm) has also announced that two abstracts involving clinical research of arimoclomol for the treatment of Niemann-Pick disease type C (NPC) have been accepted for presentation at WORLDSymposium 2023. Read the announcement.
02/23/2023
KemPharm Reports Third Quarter 2022 Results
KemPharm has shared the following news release with the NNPDF. Read news release.
11/11/2022
Orphazyme to sell to KemPharm, Inc.
NNPDF has received the following news from Orphazyme: Orphazyme A/S under In-Court-Restructuring to sell substantially all of its assets and business activities to KemPharm, Inc.
Read Orphazyme press release.
Read KemPharm press release.
We will continue to keep you informed as more information is available. If you have any questions, please contact Joslyn Crowe, NNPDF Executive Director, at jcrowe@nnpdf.org.
05/16/2022
Orphazyme Update: Orphazyme announces update on in-court restructuring proceedings
NNPDF has received the following press release which speaks to the progress the team has made in finding a potential path forward for arimoclomol.
05/03/2022
Update from Orphazyme
NNPDF has received the communication below from Orphazyme regarding this week’s press release on arimoclomol programs for NPC.
Read the letter from Orphazyme
Read the press release
03/22/2022
INPDA Letter on Arimoclomol to CHMP Members
The International Niemann-Pick Disease Alliance (INPDA) sent the following letter to the CHMP members at the European Medicines Agency (EMA) in advance of the upcoming review of Arimoclomol.
Thank you to the nearly 500 family members from around the world who signed their support as well! Read the letter.
03/17/2022
Orphazyme announces update on regulatory review of arimoclomol in the European Union
02/23/2022
Orphazyme provides update for planned NDA resubmission for arimoclomol for the treatment of Niemann-Pick disease type C in the United States