For many babies with Niemann-Pick disease type C (NPC), the first sign is in the belly. The liver and spleen are larger than they should be, and some newborns have jaundice that lingers for weeks, according to GeneReviews, the clinical reference hosted by the National Library of Medicine. Floppy muscles, missed milestones and trouble moving the eyes up and down can follow months or years later, once the disease reaches the brain.
Few treatments reach a child that young. Miplyffa is approved for children 2 and older, Aqneursa for children who weigh at least 15 kg (about 33 pounds), and every NPC treatment trial recruiting on ClinicalTrials.gov starts at age 4. On November 17, 2026 the FDA is due to decide on adrabetadex, which Beren Therapeutics has asked the agency to approve for infantile-onset NPC, meaning neurological symptoms that begin before age 6.
Which Niemann-Pick Type C Drugs Can a Baby or Toddler Get in 2026?
Under their FDA labels, neither approved NPC drug covers a typical child under 2. Miplyffa (arimoclomol), approved on September 20, 2024, is labeled for the neurological symptoms of NPC in people 2 and older and is taken together with miglustat, a pill the FDA has approved for type 1 Gaucher disease in adults and, as Opfolda, for late-onset Pompe disease, but not on its own for NPC. Aqneursa (levacetylleucine), approved on September 24, 2024, is labeled by weight from 15 kg, so whether a child qualifies depends on the scale rather than the birthday.
- Miplyffa with miglustatFDA-approved September 2024
- AqneursaFDA-approved September 2024
- Recruiting NPC treatment trialsIntraBio and Azafaros, 3 registry records
- AdrabetadexFDA decision due November 17, 2026
A child with NPC who is younger than 2 and weighs less than 15 kg falls outside both FDA-approved NPC drugs and every NPC treatment trial recruiting on ClinicalTrials.gov, which start at age 4. Trial Friend combined the 2 FDA labels with the eligibility rules of the 3 recruiting NPC treatment trial records, checked October 14, 2026.
That gap comes from Trial Friend's own check. We read the age and weight limits in the Miplyffa and Aqneursa labels on DailyMed and the minimum age of each NPC treatment trial listed as recruiting on ClinicalTrials.gov on October 14, 2026, which were IntraBio's Phase 3 study of N-acetyl-L-leucine (NCT05163288) and Azafaros's Phase 3 nizubaglustat program, registered as NCT07082725 for NPC and NCT07054515 for NPC and 2 related diseases. Each record requires a child to be at least 4. The live list is on our Niemann-Pick type C trials page.
What Infantile-Onset Niemann-Pick Type C Looks Like From Birth to Age 6
Doctors group NPC by the age at which neurological symptoms begin. GeneReviews describes an early-infantile form, starting before age 2, in which babies may have liver disease from birth, weak muscle tone and delayed development, and a late-infantile form, from 2 to under 6, in which delayed development and clumsiness often come before vertical supranuclear gaze palsy, a loss of the ability to look up and down on purpose.
Earlier brain involvement usually means faster progression. Beren cites average ages at death in past groups of children of about 5.6 years when neurological symptoms started before age 2 and about 13.4 years when they started between 2 and 6. Averages describe groups of children, and a child's own specialist is the person to ask where that child stands.
How Adrabetadex Is Given, Into the Spinal Fluid Every 2 Weeks
Adrabetadex is a mixture of cyclodextrins, ring-shaped sugar molecules that can draw cholesterol out of the compartments where it gets trapped in NPC cells, according to Beren. Because NPC damages the brain, the drug goes into the spinal fluid rather than a vein, and in its Phase 2b/3 trial it was given by lumbar puncture every 2 weeks at doses of 900 to 1800 mg, according to the study's ClinicalTrials.gov record.
We found no dose or schedule for infants in Beren's public announcements. An FDA label, if one is issued, would set both, along with which children are covered.
Does Adrabetadex Work in Infantile-Onset NPC? What Beren's Data Show
Beren's application rests mainly on comparisons with untreated children rather than a new placebo-controlled trial. Beren reported in October 2025, and repeated in March 2026, that 72 people whose neurological symptoms began before age 6 and who were treated with adrabetadex, in a Phase 2b/3 trial, its extension or expanded access, had a 71% lower risk of death than 119 matched untreated people drawn from the NIH natural history study and other databases, with 5-year survival of 84% against 42%.
A second analysis, presented in March 2026, compared 79 treated patients with themselves. Their neurological decline, scored on walking, speech, fine motor skills and swallowing, was 43% slower in the year after starting adrabetadex than before it, Beren reported.
Comparisons like these, called external controls, take the comparison group from records instead of randomly assigning children to treatment. In a disease this rare they can be the only practical option, though the FDA has to judge how closely the 2 groups match, since differences in care or in how sick the children were can widen or shrink the gap.
The only randomized trial of adrabetadex showed no difference from sham. In that Phase 2b/3 trial (NCT02534844), 56 children and young adults aged 4 to 21 received adrabetadex or a sham procedure, and after 52 weeks neither group had moved on the NPC severity score, a change of 0.0 on adrabetadex against minus 0.1 on sham, according to results posted on ClinicalTrials.gov. The trial did not enroll children under 4. Mallinckrodt, which owned the drug at the time, stopped developing it in January 2021, saying there was "no clear evidence" that it changed or improved disease progression across the patients studied, as reported by Niemann-Pick UK. Mandos, now part of Beren, bought the program that July.
Beren reads its newer analyses differently. Its chief medical officer, Alex Gold, said in March 2026 that if approved, adrabetadex would be the first option "designed to target the underlying pathophysiology of NPC", The FDA granted adrabetadex Breakthrough Therapy designation in December 2025, according to Beren, which notes that the agency had first granted it in 2016 and withdrawn it after the Phase 2b/3 trial.
Adrabetadex Side Effects, Including Hearing Loss
Hearing loss leads the list of main side effects Beren gives for adrabetadex, which it says can be managed with hearing aids, followed by tiredness or unsteadiness after a dose. The NIH reported in 2017 that patients in its early study had additional hearing loss that hearing aids compensated for.
Repeated lumbar punctures carry their own burden for a small child, with a procedure every 2 weeks that may involve sedation. Ask the care team how hearing will be tested before and during treatment and how the procedures would be scheduled.
One email when the decision is public, with what it means in plain language, plus new trials and FDA news for the condition you pick. Unsubscribe anytime.
You'll get new recruiting trials, changes to trials, FDA decisions and condition news for Niemann-Pick Disease Type C. Turn any of these off from the link in every email.
What the FDA Can Decide on Adrabetadex by November 17th
November 17, 2026 is a goal date, and the FDA can act before it. The agency moved it from August 17 after classifying Beren's March 18 response to an information request as a major amendment, Beren said in May. An approval would come with a label stating which children are covered, which may be narrower than Beren's request, while a complete response letter would mean the FDA wants more before it can approve. Breakthrough Therapy designation and priority review speed up a review without deciding its outcome.
Getting a Niemann-Pick Type C Diagnosis and Care Plan in Place Now
NPC is confirmed by finding 2 disease-causing variants in the NPC1 or NPC2 gene, and blood tests for oxysterols, cholesterol byproducts that build up in NPC, are a common first screen, according to GeneReviews. NPC is not on the federal Recommended Uniform Screening Panel for newborns, and the National Niemann-Pick Disease Foundation points to ScreenPlus, a research pilot at New York hospitals, as the newborn screening program that includes it.
Beren started a program in September 2026, called Test for NPC, that offers no-cost genome sequencing through GeneDx to eligible families, according to the company, which also estimates that about two-thirds of Americans with NPC are undiagnosed. Ask the child's doctor or a genetic counselor to go over any result.
Support is available beyond the drug decision. The National Niemann-Pick Disease Foundation has family resources and a page on Beren's program, and the NIH runs a long-running observational NPC study (NCT00344331) that accepts patients of any age, including infants, with visits at the NIH Clinical Center.
What Parents Ask About Adrabetadex and Niemann-Pick Type C
When will the FDA decide on adrabetadex?
The FDA's goal date is November 17, 2026, moved from August 17 after a major amendment. The FDA accepted Beren's application in February 2026 with priority review, and the adrabetadex FDA decision page will show the outcome the day it is public.
Can a 1-year-old take Miplyffa for Niemann-Pick type C?
Not under its FDA label, which covers people 2 and older and pairs Miplyffa with miglustat. Zevra, which makes Miplyffa, also lists an expanded access program for arimoclomol on ClinicalTrials.gov (NCT04316637), and it starts at age 2 as well.
Does adrabetadex cause hearing loss?
Hearing loss is the main side effect Beren lists for adrabetadex, and Beren says it can be managed with hearing aids. A hearing test before the first dose gives the care team a starting point to compare against later.
Is there a clinical trial for babies with Niemann-Pick type C?
No NPC treatment trial recruiting on ClinicalTrials.gov accepted children under 4 as of October 14, 2026. Observational studies do, including the NIH's NPC natural history study, which accepts any age. ScreenPlus, a newborn screening study at 9 New York hospitals, screens babies born there rather than enrolling children already diagnosed.
What is infantile-onset Niemann-Pick type C?
Beren uses the term for NPC whose neurological symptoms begin before age 6, split into early-infantile before age 2 and late-infantile from 2 to under 6. GeneReviews notes that some babies with NPC show signs even before birth, such as fluid buildup, or have liver disease as newborns.