September 26, 2024 – Just days after the FDA approved the primary treatment for a rare genetic disorder approved one other treatment for a similar condition.
The condition referred to as Niemann-Pick disease type C (NPC) is estimated to affect roughly 1 per million people within the United States. It is usually diagnosed in childhood and may cause problems with walking, speaking, swallowing, hand movements, vision and tremors. Neurological damage will be severe and the common life expectancy of patients is about 13 years.
“This is the second treatment the FDA has approved for NPC in a week. Today’s action underscores the agency’s commitment to supporting the development of new treatments for rare diseases,” said Janet Maynard, MD, MHS, director of the FDA’s Office of Rare Diseases, Pediatrics, Urology, and Reproductive Medicine, in a press release. “This approval further demonstrates FDA’s commitment to working with the scientific community to address the unique challenges that can arise in the development of medicines for rare diseases.”
The latest treatment is meant to assist improve neurological symptoms in each adults and youngsters with the disease. This happens as a result of changes within the NPC1 or NPC2 gene, which cause fats and cholesterol to get stuck in cells and damage the brain, liver and other organs.
The generic name for the brand new drug is Levacetylleucine and it's marketed under the brand name Aqneursa. The approval followed a phase III clinical trial published in January New England Journal of Medicine. The study involved 60 patients with neurological symptoms, each children (from 4 years old) and adults. The results showed that patients who took Aqneursa for 12 weeks had noticeable improvements of their ability to walk, sit, balance and speak in comparison with those that took a placebo.
In addition to the approval, the FDA also granted Aqneursa priority review and orphan drug designation, meaning it is taken into account a very important treatment for a rare disease.
Mallory Factor, CEO of IntraBio Inc., the corporate that makes Aqneursa, celebrated the approval as a major step forward in helping NPC patients. He said the corporate will make the treatment available to all individuals who need it.
Common uncomfortable side effects include stomach pain, difficulty swallowing, respiratory infections and vomiting. The FDA also warned that Aqneursa could harm unborn children. Therefore, women should inform their doctor in the event that they are pregnant or plan to turn into pregnant while using this medicine.
The FDA approved the primary drug to treat Niemann-Pick disease type C late last week. This drug, known by the brand name Miplyffa, has been approved for people ages 2 and older and shall be available in the approaching months.