FDA Approval & Clinical Milestone Pathway Simulator

Trial & Regulatory Parameters Live Sync

Pivotal Patient Cohort Size (N) 150 patients

Preclinical / Phase 2 Efficacy Score 85%

Pivotal Primary Endpoint Met

Statistically significant motor function delta

Breakthrough Therapy Designation

Expedited cross-disciplinary guidance

FDA Priority Review Voucher / Status

Accelerates target review to ~6 months

Approval Probability

92.4%

High Confidence

Estimated FDA Review

6.2 mo

PDUFA Target: 6 mo

Regulatory Status

FDA Approved (Priority Review)

Expedited Track

Milestone Rating

High Clinical Impact

Commercial Launch Ready

Bayesian Regulatory Approval Density

Posterior distribution based on cohort power, endpoint significance, and expedited FDA status

Mean: 92.4% | CI: [88.1% - 95.8%]

Calculated cycle: IND to PDUFA Target

MONTH 0 - 12

Preclinical & IND

Target validation in neuromuscular model (85% efficacy). Fast-track eligibility confirmed.

MONTH 12 - 28

Pivotal Phase 3 Trial

150-patient cohort randomized. Primary motor endpoint met with p < 0.001.

MONTH 28 - 34

BLA Submission

Biologics License Application submitted with Breakthrough Therapy designation.

MONTH 34 - 40.2

FDA Action & Nod

6.2 months Priority Review. First FDA nod granted for rare muscle-wasting indication.

Real-time FDA CDER criteria trace

[00:00.00] INIT: Calibrated model to Reuters report on Scholar Rock rare muscle-wasting therapy nod.

[00:00.01] PARAM: Cohort N=150 | Preclinical score=85 | Endpoint=PASS | Priority=TRUE | Breakthrough=TRUE

[00:00.02] DECISION: Expedited 6-month review granted under PDUFA VII guidelines. Posterior Approval: 92.4%.