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.
Top Events Near-Term (Next 2 Weeks)
Date: Nov 30
Event: PDUFA (Achondroplasia)
PoA: 80
Setup: FDA accepted the NDA for TransCon CNP with Priority Review. The pivotal ApproaCH trial showed a statistically significant improvement in annualized growth velocity (AGV) compared to placebo in children with achondroplasia.
Our View: High PoA. Strong placebo-controlled efficacy and a clean safety profile. The key watch item is the label, especially growth-velocity language and positioning versus BioMarin's Voxzogo.
Avance® Nerve Graft — $AXGN ( ▲ 1.98% )
Date: Dec 5
Event: PDUFA (Biologic BLA)
PoA: 72
Setup: The FDA extended the PDUFA date to December 5 after accepting a Major Amendment to review additional manufacturing and facility information.
Our View: This is primarily a CMC/quality binary, not a clinical one. Base case is procedural delay and eventual approval; bear case is deeper consistency or facility concerns.
ORLADEYO (berotralstat, oral granules) — $BCRX ( ▲ 1.05% )
Date: Dec 12
Event: PDUFA (Pediatric HAE, 2–11 years)
PoA: 86
Setup: The pediatric NDA was accepted with Priority Review. The current Dec 12 action date reflects a 3-month PDUFA extension from the original September 12 date after BioCryst submitted additional formulation data in June, which FDA classified as a major amendment requiring full review.
Our View: Very high PoA. If approved, ORLADEYO becomes the first oral targeted prophylactic for HAE in children under 12, strengthening the pipeline against injectable competitors.
📆 Event Update (Clarified Timelines)
Bezuclastinib — $COGT ( ▼ 2.47% )
PEAK (2L GIST) Phase 3 Topline → Resolved (strongly positive, Nov 10).
APEX (Advanced Systemic Mastocytosis) → Topline expected December 2025, not November.
Will appear in a future Pulse once date is confirmed.
Nimacimab — $SKYE ( ▼ 2.5% )
Phase 2a topline reported Oct 6: monotherapy failed the primary endpoint (p=0.2699).
GLP-1 combination arm remains the only viable bull case.
Not a November binary.
✅ Resolved Last Week (Nov 17–23)
Plozasiran (REDEMPLO) — $ARWR ( ▼ 0.77% )
Event: PDUFA (FCS)
Outcome: Approved (Nov 18)
Context: FDA approved plozasiran as the first siRNA therapy for familial chylomicronemia syndrome. Phase 3 data showed ~80% median triglyceride reduction and reduced pancreatitis incidence. The market now focuses on pricing, access, and competitive dynamics.
Ziftomenib (KOMZIFTI) — $KURA ( ▼ 1.69% )
Event: PDUFA (originally Nov 30)
Outcome: Approved Early (Nov 13)
Context: Approved for adults with relapsed/refractory AML harboring an NPM1 mutation. The label includes a Boxed Warning for differentiation syndrome — the key gating issue in the regulatory debate. Efficacy is validated; commercial risk centers on DS management and adoption in real-world oncology settings.
Mitapivat (PYRUKYND) — $AGIO ( ▼ 3.07% )
Event: Phase 3 Topline — RISE UP
Outcome: Mixed (Nov 19)
Context: Met the hemoglobin-response primary endpoint and key hemolysis secondaries, but failed the co-primary endpoint of annual sickle cell pain crises and failed the PROMIS fatigue endpoint. Biology is intact; the commercial thesis took a hit. The 50% pre-data drawdown now looks like informed de-risking around the trial’s ability to meet the crises endpoint.
📊 PoA Framework (Snapshot)
PoA Bands:
High: >70
Moderate: 40–70
Speculative: <40
Five-Factor Model:
Study design
Data strength
Safety / CMC
Regulatory precedent
Sponsor execution
Directional only; always pair PoA with context on trial design, division history, and tape behavior.