September 24, 2024 – The FDA has approved the primary treatment for a rare genetic disorder, often diagnosed in childhood, that may damage the nerves and brain and result in organ failure over time. People with this condition live about 13 years on average.
It is estimated that fewer than 1,000 people within the United States are affected by Niemann-Pick disease type C, which increasingly affects the flexibility to talk, swallow and walk or move independently.
The disease, which also affects mental functions resembling learning and memory, is a Lysosomal storage disorder – characterised by a buildup of fatty substances resembling cholesterol that damage the body and brain.
The generic name for the brand new drug is Arimoclomol and it's marketed under the brand name Miplyffa. It is approved for people ages 2 and older through a mixture of FDA approvals — including Fast Track and Breakthrough designations, in addition to a special program that rewards firms for brand new pediatric treatments.
Niemann-Pick disease type C, or NPC, “is a serious illness that has enormous negative impact on patients and families.” Despite extensive research efforts, there aren't any approved treatments that meet the numerous needs of patients,” said Janet Maynard, MD, MHS, director of the FDA's Office of Rare Diseases, Pediatrics, Urology and Reproductive Medicine, in an announcement. “The first-ever approval of a safe and effective medication option for NPC will undoubtedly meet the essential medical needs of those suffering.”
In its approval, the FDA cited a clinical trial of Miplyffa that included 50 individuals with the condition ages 2 to 19. Many participants within the study were also taking one other drug called miglustat, and the brand new FDA approval calls for the 2 drugs to be taken together. Both are taken orally.
People who took Miplyffa with miglustat for 12 months stopped disease progression as measured by a symptom severity scale, based on a study Press release from drugmaker Zevra Therapeutics.
In its approval announcement, the FDA described the treatment as resulting in “slower disease progression.” As a part of its application, Zevra Therapeutics also submitted data from a 48-month study following the initial study, the corporate said.
In 2021, the FDA declined to approve Miplyffa. But in August an FDA advisory committee voted 11 to 5 in support of an announcement that the drug has been shown to be effective.
Those who voted in favor noted that there was an unmet need for such treatment, there have been no obvious safety concerns, and the available data was consistent. Dissenters said the information “did not meet the standards of evidence” and called for extra studies, in addition to further tests on animals and in laboratories, to raised assess how the drug works, based on a summary of the meeting.
In its approval, the FDA noted that common unwanted effects of Miplyffa include upper respiratory tract infections, diarrhea, and weight reduction.
Zevra Therapeutics said the drug can be available inside 2 to three months.