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The FDA is expected to decide on ST-920 for Fabry disease in an undisclosed window, under a BLA (rolling) from PTC Therapeutics.
What the FDA is deciding on ST-920 for Fabry disease
The FDA is reviewing PTC Therapeutics' application to approve ST-920 (isaralgagene civaparvovec) for fabry disease.
One-time AAV gene therapy with RMAT designation; rolling BLA underway with the final module expected in Q4 2026. PTC completed its acquisition of the program from Sangamo on September 21, 2026. A PDUFA date follows once the FDA accepts the completed application.
Who the ST-920 decision matters to
Fabry disease is a rare inherited lysosomal storage disorder where a missing enzyme causes fatty substances called globotriaosylceramide to accumulate in cells throughout the body. This buildup damages blood vessels, organs, and tissues, leading to progressive kidney, heart, and neurological problems.
Enzyme replacement therapy, and for adults with certain gene variants a chaperone pill, can help manage the condition.
Prevalence: 1 in 40,000 to 60,000 males; higher in females when accounting for carrier status. See our full Fabry Disease page for current treatments, recruiting trials, and community resources.
What an approval or a rejection of ST-920 would mean
An approval starts a second race rather than ending the first one: specialty pharmacy setup, insurance review, and patient assistance typically take weeks even when everything goes right. Our guide to the 90 days after a rare disease approval explains the timeline and the moves families can make on day 1.
A complete response letter would mean the FDA declined to approve in the application's current form. The letter describes what the FDA wants addressed, and the company then decides whether to resubmit, run more studies or stop. Either way, this page updates with the outcome and what it means.
Where Fabry Disease treatment stands today
Read our full Fabry Disease treatment summary
Fabry disease trials today include one-time gene therapies, new pills and studies that follow people on the approved medicines. uniQure's gene therapy AMT-191 is in a recruiting Phase 1/2 trial (NCT06270316). Idorsia's lucerastat pill has a Phase 3 trial in untreated adult men, Fab-Klear (NCT07778667), that is not yet recruiting.
Amicus is testing Galafold in children aged 2 to 11 with amenable gene variants (NCT06904261). Substrate reduction pills have not yet worked in Fabry trials.
Sanofi said on February 2, 2026 that venglustat missed the main goal of its PERIDOT pain trial, and its second-quarter 2026 results, published July 30, 2026, list venglustat for Fabry disease as removed from Phase 3 because it did not meet its primary endpoint.
Cascade screening of family members, particularly female relatives, can identify others who might benefit from early care. A metabolic specialist or cardiologist can talk through which trials fit a person's disease pattern and whether they might qualify.
The Fabry Disease page has every treatment and trial →Meanwhile, 44 Fabry Disease studies are open or about to open
Whatever the FDA decides here, research on Fabry Disease does not stop. Of these, 14 test a treatment; the others follow patients over time. The most recently updated:
We'll email you when the decision is public, with what it means in plain language, and when new trials open for Fabry Disease. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
Frequently asked questions
When will the FDA decide on ST-920?
PTC Therapeutics has not disclosed a decision date.
Can it move: the FDA can act early, and a major amendment to the application can push the date back 3 months.
Source: PTC Therapeutics press release
What is ST-920 being reviewed for?
PTC Therapeutics submitted a BLA (rolling) for ST-920 in Fabry disease.
What to know: One-time AAV gene therapy with RMAT designation; rolling BLA underway with the final module expected in Q4 2026. PTC completed its acquisition of the program from Sangamo on September 21, 2026. A PDUFA date follows once the FDA accepts the completed application.
Is there already an FDA-approved treatment for Fabry Disease?
Yes. Trial Friend's drug list includes 3 FDA-approved drugs for Fabry Disease.
Approved: Fabrazyme (agalsidase beta), Galafold (migalastat), Elfabrio (pegunigalsidase alfa).
Check: the Fabry Disease page also searches FDA drug labels for approved treatments.
Can I get ST-920 before the FDA decides?
Usually only through a clinical trial or an expanded access program, and the company decides whether to offer one.
Trials: 44 Fabry Disease studies are open or about to open, 14 of them testing a treatment. See them.
Check your fit: Match Me compares your details with every open trial.
Expanded access: your doctor can ask PTC Therapeutics directly. Registered programs are listed on ClinicalTrials.gov.
How much will ST-920 cost?
PTC Therapeutics has not announced a price. Drugmakers usually set it around approval.
Help paying: drugmaker programs and charity funds usually open at launch.
Find help: the patient assistance finder lists programs by insurance type.
What happens after the ST-920 decision?
This page updates with the outcome the day it is announced.
If approved: getting the drug still takes time. Specialty pharmacy setup, insurance review and patient assistance often take weeks. Our guide covers what families can do on day 1.
If not approved: a complete response letter means the FDA declined the application in its current form. Many are about manufacturing or how data were presented, and companies often resubmit.
Where this date comes from
The FDA does not publish PDUFA dates; companies disclose them. This one comes from PTC Therapeutics press release. Dates can move, and the FDA can act early or late. This page rechecks against our calendar, which is re-verified daily.