PDUFA Dates September 2026: 11 FDA Decisions on the Calendar

Update September 4, 2026: The Ionis zilganersen PDUFA resolved early — approved September 3 as ZANVASTRO (see our approval coverage).

Update September 24, 2026: Four more September dates resolved: Telix’s Pixclara (approved Sep 14), Ultragenyx’s UX111 (approved Sep 17 as Fayuvi), Elevar’s lirafugratinib (approved Sep 23 as Lyrfigtu), and Scholar Rock’s apitegromab (approved Sep 11 as Isembyld). Still ahead: Egetis Emcitate (Sep 28) and BMY Camzyos adolescent sNDA (Sep 30).

Update September 26, 2026: Mirum’s Atebrioz (zilurgisertib) was approved September 25 for FOP in patients 12 and older — one day ahead of its September 26 PDUFA (see our Atebrioz coverage). Two September dates remain: Egetis Emcitate (Sep 28) and BMY Camzyos adolescent sNDA (Sep 30).

Update September 28, 2026: Egetis’ Emcitate (tiratricol) was approved September 28 for peripheral thyrotoxicosis in MCT8 deficiency (Allan-Herndon-Dudley syndrome), the first-ever FDA-approved treatment for the ultra-rare disorder (novel drug #45 of 2026). Egetis trades on Nasdaq Stockholm (EGTX), not a US listing, so there is no BBS ticker. Only BMY’s Camzyos adolescent sNDA (Sep 30) remains on the September calendar.

September is the busiest PDUFA month of the second half: 11 FDA target action dates across rare disease, oncology, neurology, and hematology, ranging from binary small-cap events to mega-cap label expansions. Here is the full calendar, ranked by how much each decision can actually move the underlying stock. We track all of these live on the FDA calendar page.

The calendar at a glance

Date	Company (Ticker)	Drug	Application	Indication
Sept 3	Advicenne	Sibnayal	NDA	Distal renal tubular acidosis
Sept 11	Telix (TLX)	Pixclara (TLX101-Px)	NDA	CNS tumor imaging — Approved Sep 14
Sept 18	Nuvalent/GSK	Zidesamtinib	NDA	ROS1+ NSCLC (approved early — see below)
Sept 19	Ultragenyx (RARE)	Fayuvi (UX111)	BLA	Sanfilippo syndrome type A — Approved Sep 17
Sept 21	Merck (MRK)	Winrevair	BLA	Pulmonary arterial hypertension — Label update approved Sep 22
Sept 22	Ionis (IONS)	Zilganersen	NDA	Alexander disease — Approved Sep 3
Sept 26	Incyte/Mirum (MIRM)	Atebrioz (zilurgisertib)	NDA	Fibrodysplasia ossificans progressiva — Approved Sep 25
Sept 27	Elevar	Lyrfigtu (lirafugratinib)	NDA	FGFR2+ cholangiocarcinoma — Approved Sep 23
Sept 27	Praxis (PRAX)	Relutrigine	NDA	SCN2A/SCN8A DEEs — moved to Dec 27
Sept 28	Egetis	Emcitate (tiratricol)	NDA	MCT8 deficiency — Approved Sep 28
Sept 30	ScholarRock (SRRK)	Isembyld (apitegromab)	BLA	Spinal muscular atrophy — Approved Sep 11
Sept 30	Bristol Myers Squibb (BMY)	Camzyos sNDA	sNDA	Adolescent oHCM
Sept (TBD)	Roivant/Priovant	Brepocitinib (LISRAYA)	NDA	Dermatomyositis — Approved Aug 27
Sept (TBD)	Takeda/Protagonist	Rusfertide (MIMRYLO)	NDA	Polycythemia vera — Approved Aug 28

Nine September entries have now resolved: Nuvalent’s zidesamtinib was approved early on July 22 as Jideytro after GSK’s $10.6 billion acquisition closed, Takeda’s Orzeyful (oveporexton) narcolepsy approval landed August 5, Roivant’s brepocitinib (LISRAYA) was approved August 27 for dermatomyositis — five weeks ahead of its window — and Takeda/Protagonist’s rusfertide (MIMRYLO) was approved August 28 for polycythemia vera. See our MIMRYLO coverage. Since then, four more resolved: Telix’s Pixclara (September 14), Ultragenyx’s UX111 (September 17 as Fayuvi), Elevar’s lirafugratinib (September 23 as Lyrfigtu), and Scholar Rock’s apitegromab (September 11 as Isembyld). Mirum’s Atebrioz (zilurgisertib) was approved September 25 for FOP, one day ahead of its September 26 PDUFA. Egetis’ Emcitate (tiratricol) was approved September 28 for MCT8 deficiency, the last small-cap September date to resolve.

The three decisions that can move stocks 30%+

Ultragenyx UX111, September 19 (BLA resubmission, gene therapy) — RESOLVED. Approved September 17 as Fayuvi (rebisufligene etisparvovec), the first-ever treatment for Sanfilippo syndrome type A. The FDA granted full approval. Full thesis in our UX111 deep dive.

Praxis Relutrigine, December 27 (was September 27 — extended). Praxis Precision Medicines (PRAX) filed relutrigine for SCN2A and SCN8A developmental and epileptic encephalopathies — severe pediatric seizure disorders with no approved therapies for most patients. The FDA extended the review period by three months in June, pushing the PDUFA from September 27 to December 27, 2026, after Praxis submitted additional sensitivity analyses the agency deemed a major amendment. See our relutrigine primer.

ScholarRock Apitegromab, September 30 (BLA, Priority Review) — RESOLVED. Approved September 11 as Isembyld (apitegromab-mstn), the first muscle-targeted add-on to SMN therapy for spinal muscular atrophy — roughly 19 days ahead of its PDUFA. See our apitegromab primer.

The rest, ranked by materiality

Ionis Zilganersen, September 22 (NDA, Priority Review) — RESOLVED. Approved September 3 as ZANVASTRO, the first disease-modifying treatment for Alexander disease (see our approval coverage). The platform story matters more than any single rare disease NDA, but the approval adds a second approved ASO in 2026 and validates the neuro pipeline.

Mirum/Incyte Zilurgisertib, September 26 — RESOLVED. Approved September 25 as Atebrioz, one day ahead of its PDUFA and roughly five weeks after Regeneron’s Pasatru approval in the same disease on August 19 (see our Pasatru analysis and Atebrioz coverage). Two FOP drugs in five weeks — now three total — for a disease with 800 U.S. patients. Competitive dynamics will decide which wins the small market. Primer: Mirum zilurgisertib.

Elevar Lirafugratinib, September 27 — RESOLVED. Approved September 23 as Lyrfigtu for FGFR2+ cholangiocarcinoma — Elevar’s first US approval. Our primer.

BMY Camzyos adolescent sNDA, September 30. Label expansion for symptomatic obstructive hypertrophic cardiomyopathy in adolescents. Immaterial to BMY’s ~$110 billion cap but strategically extends the franchise before competitors arrive.

Merck Winrevair sBLA, September 21 — RESOLVED. The pulmonary arterial hypertension label expansion was approved September 22 (HYPERION and ZENITH trial data added to the label). Winrevair is one of 2025-2026’s fastest-growing launches. For context on why PAH is suddenly crowded, see the Sotatercept competitive landscape coverage in our FDA calendar archive.

Egetis Emcitate (Sept 28), Advicenne Sibnayal (Sept 3), Telix Pixclara (Sept 11). Pixclara was approved September 14 as the first FET-PET imaging drug for glioma, and Emcitate was approved September 28 for MCT8 deficiency (novel drug #45 of 2026, first-ever US treatment for the disorder). Sibnayal remains — an ultra-rare play at a small sponsor with minimal index impact.

Roivant/Priovant Brepocitinib (LISRAYA) — APPROVED August 27. A TYK2/JAK1 inhibitor for dermatomyositis, cleared roughly five weeks ahead of its September 30 window as the first targeted therapy for the disease. See our LISRAYA coverage.

How to trade the month

September’s calendar is dominated by rare disease filings with strong biomarker data and Priority Review — historically the highest-approval-rate category in PDUFA outcomes. The pattern that repeats: the run-up arrives in the two weeks before the date, and the decision-day move depends on how much of the approval was already priced. UX111 has been the market’s known September trade since the BLA resubmission; expect crowded positioning. With Mirum, Elevar, and Egetis Emcitate now resolved and Praxis moved to December 27, the only September date still open is BMY’s Camzyos adolescent sNDA (Sep 30) — immaterial to BMY’s ~$110 billion cap. For the full framework — entry timing, AdCom reads, exit discipline — see how to trade FDA catalysts and what a PDUFA date is.

We will update each decision live on the FDA calendar as dates resolve.

Sources: Cardiology Advisor September PDUFA roundup | Assyro PDUFA Calendar 2026 | checkrare 2026 orphan drug PDUFA dates