September 24, 2024 – The FDA has approved the primary treatment for a rare genetic disorder, often diagnosed in childhood, that could cause nerve and brain damage and, over time, result in organ failure. People with the disease live a median of about 13 years.
It is estimated that fewer than 1,000 people within the United States are affected by Niemann-Pick disease type C. It progressively impairs the flexibility to talk, swallow, and walk or move independently.
The disease, which also affects mental functions comparable to learning and memory, is a lysosomal storage disease – characterised by an accumulation of fatty substances comparable to cholesterol, which damage the body and brain.
The recent drug's generic name 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 processes – including fast-track and breakthrough approvals, in addition to a special program that rewards corporations for brand new treatments for kids.
Niemann-Pick disease type C, or NPC, “is a serious illness that has a tremendous negative impact on patients and families. Despite extensive research efforts, there are no approved treatments that address the significant needs of patients,” Janet Maynard, MD, MHS, director of the FDA's Office of Rare Diseases, Pediatrics, Urology and Reproductive Medicine, said in an announcement. “The first-ever approval of a safe and effective drug option for NPC will undoubtedly address the essential medical needs of those affected.”
In its approval, the FDA cited a clinical trial of Miplyffa that involved 50 people between the ages of two and 19. Many of the study participants also took one other drug called miglustat, and the brand new FDA approval calls for the 2 drugs to be taken together. Both are taken orally.
In individuals who took Miplyffa with miglustat for 12 months, the progression of the disease was stopped, as measured by a symptom severity scale. Press release from the drug manufacturer Zevra Therapeutics.
In its approval statement, the FDA described the treatment as “leading to slower disease progression.” As a part of its application, Zevra Therapeutics also submitted data from a 48-month study following the primary trial, the corporate said.
In 2021, the FDA rejected approval of Miplyffa. But in August, an FDA advisory committee decided Vote 11-5 to support the claim that the drug has been proven effective.
Those who voted in favor identified that there's an unmet need for such a treatment, there are not any obvious safety concerns and the available data is consistent. Opponents of the vote said the info “did not meet the standards of evidence” and called for further studies and more testing on animals and in laboratories to higher assess the drug's effects, in line with a summary of the meeting.
In its approval, the FDA noted that common unwanted side effects of Miplyffa include upper respiratory tract infections, diarrhea, and weight reduction.
Zevra Therapeutics announced that the drug could be available inside 2 to three months.