SILVER
SPRING, Md., Sept. 20,
2024 /PRNewswire/ -- Today, the U.S. Food and
Drug Administration approved Miplyffa (arimoclomol), an oral
medication for the treatment of Niemann-Pick disease, type C (NPC).
Miplyffa, in combination with the enzyme inhibitor miglustat, is
approved to treat neurological symptoms associated with NPC in
adults and children 2 years of age and older. Miplyffa is the first
drug approved by the FDA to treat NPC.
NPC is a rare genetic disease that results in progressive
neurological symptoms and organ dysfunction. It is caused by
changes in either the NPC1 or NPC2 gene, affecting
the necessary transport of cholesterol and other lipids within a
cell. As a result, these cells do not function as they should,
ultimately causing organ damage. On average, individuals affected
by this devastating disease only live for about 13 years.
"NPC is a serious disease that leads to enormous adverse
impacts on patients and families. Despite extensive research
efforts, there have not been approved treatments to meet the
significant needs of patients," said Janet
Maynard, M.D., M.H.S., director of the Office of Rare
Diseases, Pediatrics, Urologic and Reproductive Medicine (ORPURM),
in the FDA's Center for Drug Evaluation and Research. "The
first-ever approval of a safe and effective drug option for NPC
will undoubtedly support the essential medical needs of those
suffering."
Miplyffa was the first product application to be discussed at
the inaugural meeting of the Genetic Metabolic Diseases Advisory
Committee (GeMDAC) in August. GeMDAC was established in
December 2023 to advise the agency on
products used for the diagnosis, prevention or treatment of genetic
metabolic diseases.
The safety and effectiveness of Miplyffa were evaluated in
a randomized, double-blind, placebo-controlled 12-month trial in
patients two to 19 years of age who had a molecularly confirmed
diagnosis of NPC. Fifty patients were randomized 2:1 to treatment
with weight-adjusted Miplyffa (31 to 124 mg) or placebo orally
three times per day. Among these 50 patients, 39 (78%) received
miglustat as background treatment in the trial.
Miplyffa's efficacy was demonstrated by the rescored 4-domain
NPC Clinical Severity Scale (R4DNPCCSS) score in the patients who
used miglustat as their background treatment. The R4DNPCCSS is a
measure of NPC disease progression that looks at four items that
patients with NPC, their caregivers and physicians have identified
as most relevant including ambulation, speech, swallow and fine
motor skills. Higher scores signify a greater severity of the
disease. Compared to placebo, Miplyffa resulted in a slower disease
progression as measured by the R4DNPCCSS score.
The prescribing information for Miplyffa contains a warning for
hypersensitivity reactions including hives and angioedema (swelling
under the skin). Individuals experiencing these adverse reactions
should stop using the drug. Females who are pregnant or plan to
become pregnant should not use Miplyffa.
The most common side effects of Miplyffa include upper
respiratory tract infection, diarrhea and decreased weight.
Miplyffa, along with miglustat, should be taken orally with or
without food according to the recommended dose for the patient's
body weight.
The FDA granted Miplyffa priority review, orphan drug, rare
pediatric disease, fast track and breakthrough therapy designations
for this application.
The FDA granted approval of Miplyffa to Zevra Therapeutics.
Additional Resources:
- August 2, 2024 Meeting of the
Genetic Metabolic Diseases Advisory Committee
- Office of Rare Diseases, Pediatrics, Urologic and Reproductive
Medicine (ORPURM)
Media Contact: April
Grant, 202-657-8179
Consumer Inquiries: Email or 888-INFO-FDA
The FDA, an agency within the U.S. Department
of Health and Human Services, protects the public health by
assuring the safety, effectiveness, and security of human and
veterinary drugs, vaccines and other biological products for human
use, and medical devices. The agency also is responsible for the
safety and security of our nation's food supply, cosmetics, dietary
supplements, products that give off electronic radiation, and for
regulating tobacco products.
View original content to download
multimedia:https://www.prnewswire.com/news-releases/fda-approves-first-treatment-for-niemann-pick-disease-type-c-302254310.html
SOURCE U.S. Food and Drug Administration