PoA = directional probability-of-approval estimate based on public data, historical precedent, and market context. These are internal analytical views — not FDA guidance or statistical predictions.

Date: Dec 5
Event: PDUFA (Biologic BLA)
PoA: 72

Setup:
The FDA extended the PDUFA date to December 5 after classifying newly submitted manufacturing and facility documentation as a major amendment. Long-term RANGER registry data continue to support clinical performance in peripheral nerve repair, with labeling feedback expected imminently.

Our View:
This remains a CMC/quality-driven review rather than a clinical hurdle. Base case is approval with labeling refinements; key risks center on facility inspections, but precedent favors procedural resolution.

Date: Dec 2
Event: Full Data Presentation (ZIRCON-X, SUO Annual Meeting)
PoA: 75

Setup:
Topline results for the ZIRCON-X extended analysis of the Phase 3 ZIRCON trial were released on November 20, demonstrating a 48.6% change in surgical management for indeterminate renal masses. The December 2 event corresponds to detailed data presentation at the Society of Urologic Oncology (SUO) meeting in Phoenix.

Our View:
Diagnostic performance continues to look strong. Formal conference data could accelerate BLA preparation and reinforce Telix’s expanding urologic oncology franchise alongside Illuccix. Specificity and management-change metrics remain the key watchpoints.

Date: Dec 7
Event: PDUFA (TD/NTD Thalassemia)
PoA: 80

Setup:
The sNDA review was extended three months to December 7 following REMS submission addressing hepatocellular injury risk. ENERGIZE trials showed meaningful hemoglobin improvements in alpha/beta-thalassemia. EMA CHMP issued a positive opinion in October.

Our View:
High PoA with REMS implementation de-risking safety concerns. Approval would expand PYRUKYND beyond PKD into a ~30K-patient U.S. thalassemia population with limited oral treatment options.

Mitapivat (PYRUKYND) — $AGIO ( ▼ 3.07% )
RISE UP Phase 3 topline (sickle cell) → Resolved (mixed, Nov 19). Met hemoglobin endpoint but missed pain crises. Biological activity remains validated; no impact on the Dec 7 thalassemia action date.

Buntanetap — $ANVS ( ▼ 2.78% )
Full biomarker presentations at CTAD (Dec 1–4).
Company schedules indicate both the Alzheimer’s (Phase 2/3 design + biomarkers) and Parkinson’s (Phase 3 biomarker set) datasets will be presented during the Dec 1–2 window.
Note: Topline Parkinson’s biomarker/cognitive data was released earlier on Nov 17; CTAD reflects expanded data, not a fresh readout.

Event: PDUFA (Achondroplasia)
Outcome: Review Extended — New Date Feb 28, 2026

Context:
The FDA issued a three-month extension after a major amendment related to post-marketing study protocols. ApproaCH trial efficacy remains intact. The delay is procedural rather than a clinical setback, shifting focus to Q1 2026 labeling negotiations versus Voxzogo.

PoA Bands:

Five-Factor Model:

Directional only. Always pair PoA with context on trial design, division history, and tape behavior.