Agios Pharmaceuticals
Agios Pharmaceuticals
One-line summary: Rare-disease biotech (NASDAQ: AGIO); mitapivat sNDA accepted for FDA Priority Review in sickle cell after a missed SCPC/VOC co-primary — the wiki's FDA rare-disease right-shift acceptance datapoint. PDUFA November 1, 2026 CONFIRMED. Approval has not happened. "No ADCOM" is a podcast/wiki prior, not in the July 7 or July 30 company PRs.
Valuation (August 28, 2026)
Last: $33.32 at the Friday 2026-08-28 4:00 PM ET close. From 2026-08-31-agios-agio-mitapivat-scd-pdufa-november-1 (stockanalysis AGIO as cited in the clip). 2026-08-29 is Saturday — no Friday-the-29th session.
Gage's judgment (as of 2026-08-31)
Gage, 31 Aug 2026. ~40% up / ~35% down into the Nov 1, 2026 FDA decision. Watch, not a sleeve. Gage's judgment, not grain / not issuer fact. Last-price as-of stays the vault last: $33.32 (Friday 2026-08-28 4:00 PM ET). Binary on the mitapivat SCD sNDA. Approval despite missed VOC = posture holds — this is Gage's watchpoint, not grain; grain on this page still says do not file "approved despite missed VOC" — PDUFA is still Nov 1. Break = CRL or a surprise ADCOM (no-ADCOM still unverified). REIGNITE confirmatory runs into 2029. Not a buy/sell/size. Not a paper sleeve. No source URL — do not invent one.
What it is
PK-activator / rare-hematology biotech. Mitapivat (Pyrukynd® is the U.S. brand for PK deficiency; AQVESME™ for thalassemia). The sickle-cell filing is an sNDA for mitapivat, not a PYRUKYND-branded SCD sNDA — the July 7, 2026 Agios PR does not title it as a PYRUKYND sNDA. From 2026-08-31-agios-agio-mitapivat-scd-pdufa-november-1.
Why it matters to stock-market
The acceptance anchor of fda-right-shift-to-rare-disease-refile-rerate: FDA accepted the mitapivat SCD sNDA for Priority Review (accelerated pathway) after RISE UP missed its SCPC/VOC co-primary. That is the confirmable test. Do not file "approved despite missed VOC" — PDUFA is still November 1, 2026. Analysts re-rated AGIO's probability-of-success "well above 50%" on the July 10 podcast; that remains a panel read, not a company PR.
Key facts
- Ticker: AGIO (NASDAQ)
- PDUFA November 1, 2026 CONFIRMED from the July 7, 2026 Agios PR ("FDA's PDUFA goal date is November 1, 2026") and restated in the Q2 2026 earnings PR (July 30). Priority Review; accelerated-approval pathway; confirmatory Phase 3 REIGNITE. From 2026-08-31-agios-agio-mitapivat-scd-pdufa-november-1.
- Confirm test = Priority Review acceptance after missed SCPC, not approval. November 19, 2025 RISE UP topline PR: hemoglobin response met (40.6% vs 2.9%, p<0.0001); annualized SCPC missed (2.62 vs 3.05, p=0.1213). The July 7 PR describes the two primaries but does not say the pain-crisis co-primary was missed.
- "No ADCOM" is unverified from company PRs. The July 7 Priority Review PR and the July 30 Q2 PR contain zero advisory-committee / ADCOM language. The no-ADCOM sentence in this wiki came from 2026-07-10-podcast-biotech-hangout-episode-188-july-10-2026 (eric-schmidt). Leave it as a podcast/wiki prior.
- REIGNITE is underway, not stalled. Q2 PR: first patient dosed. ClinicalTrials.gov NCT07656415: status RECRUITING; start 2026-07-21; primary completion est. 2029-08. From 2026-08-31-agios-agio-mitapivat-scd-pdufa-november-1.
Breaks (not predictions)
From 2026-08-31-agios-agio-mitapivat-scd-pdufa-november-1:
- CRL (or complete-response equivalent) on the SCD sNDA.
- Surprise ADCOM — FDA/Agios notice that an AdCom will be held, or a late calendar listing before November 1.
- REIGNITE stall — hold, termination, enrollment freeze, or protocol rewrite that delays the confirmatory clock.
Sources
- 2026-08-31-agios-agio-mitapivat-scd-pdufa-november-1
- 2026-07-10-podcast-biotech-hangout-episode-188-july-10-2026