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Drug Review Profiles

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Lantidra Chronology: 19 Years From IND Submission To Approval For The Cellular Therapy

Pink Sheet’s Drug Review Profile looks at the timeline behind the development and FDA review of CellTrans’ treatment for type 1 diabetes.

Drug Review Profile Regenerative Medicine

Lantidra’s First-Cycle Complete Response Letter Centered On Inspection And Product Quality Issues

Although the CRL delayed the cell therapy’s approval by almost two years, additional CMC-related testing requested by the FDA ultimately came to be seen as a good thing by sponsor CellTrans because it provided assurance of product consistency.

Drug Review Profile Complete Response Letters

‘Poor Quality’ Submission: CellTrans’ Lantidra BLA Marked By Missing And Incongruent Data

FDA's review of documents and source data files to assess product efficacy and subject safety was complicated by missing and inconsistent data, the clinical reviewer said, concluding there was insufficient data monitoring during clinical studies or in preparing the documents for regulatory submission.

Drug Review Profile Regenerative Medicine

CellTrans’ Lantidra: Type 1 Diabetes Cell Therapy Overcame The Odds On Nontraditional Path To Approval

Despite a host of challenges with donislecel’s development and BLA, the US FDA approved based on clinical data from only 30 patients, using a clinically meaningful insulin independence endpoint and in a more carefully tailored population. Pink Sheet’s Drug Review Profile takes a deep dive into the FDA review of the first approved allogenic pancreas islet cell product.

Drug Review Profile Regenerative Medicine

Let’s Go To The Video: Recordings Of DMD Patients After Elevidys Treatment ‘Compelling’ But Not Substantial Evidence

CBER Director Peter Marks urged the review teams for Sarepta’s gene therapy to consider pre- and post-treatment videos showing improved physical function, but clinical and statistical reviewers cited limitations in the patient videos and said they failed to inform whether micro-dystrophin is a suitable surrogate endpoint for accelerated approval.

Drug Review Profile Approvals

Elevidys Clinical Development: Confidence In Surrogate Endpoint A Longstanding Concern For FDA Reviewers

Timeline of the gene therapy’s clinical development and regulatory review shows agency staff repeatedly questioned the ability of micro-dystrophin to predict clinical benefit in Duchenne muscular dystrophy patients.

Drug Review Profile Approvals

Sarepta’s External Controls Analysis Weakened By Elevidys Placebo Data Comparison

When placebo data from the only randomized trial of the Duchenne muscular dystrophy gene therapy were compared with Sarepta’s proffered external control data, the placebo subjects numerically outperformed the external controls, ‘calling into question the comparability of the external control group,’ an FDA statistical reviewer said.

Drug Review Profile Approvals

Elevidys’ Age-Restricted Indication Not Negotiable, CBER’s Peter Marks Told Sarepta

FDA team held several internal meetings on the gene therapy after the 12 May advisory committee, culminating in a teleconference with Sarepta in which the CBER director outlined his accelerated approval decision and reiterated recommendations to modify the ongoing EMBARK trial to better ensure confirmation of benefit. Pink Sheet's Drug Review Profile dives into the story behind the Elevidys review.

Drug Review Profile Approvals

Omisirge Review Chronology: From Pre-IND Meeting To Approval

The Pink Sheet charts the 13-year journey from Gamida Cell's initial meeting with the US FDA to Omisirge's eventual approval ahead of an extended user fee date.

Drug Review Profile Cancer

Expanding Cells, Expanding Access: Gamida Cell’s Omisirge Debuts New Donor Source For Stem Cell Transplant

Pink Sheet’s Drug Review Profile follows the FDA’s navigation of the first expanded umbilical cord blood graft with the first indication grounded in neutrophil recovery and infection incidence data.

Drug Review Profile Diversity & Inclusion

Leqembi Clinical Development: Aduhelm Opened The Door For Accelerated Approval

Timeline of lecanemab’s clinical development shows how, almost three years after telling Eisai and Biogen that their Phase II results did not support regular or accelerated approval, the FDA review division reversed course following its decision to approve aducanumab on a surrogate endpoint.

Drug Review Profile Drug Review

Leqembi Safety: Label Has Distinct MRI Schedule But Follows Aduhelm On ARIA Management

With the second amyloid-targeting antibody for treatment of Alzheimer’s, US FDA opted for a class-wide approach to management of amyloid-related imaging abnormalities, but with a product-specific MRI monitoring schedule that differs from that of Aduhelm.

Drug Review Profile Drug Review

Leqembi Phase II Missed Clinical Endpoint But Still Provided Support For Amyloid Surrogate

Pink Sheet's Drug Review Profile digs into the FDA memos on Eisai/Biogen’s lecanemab; Phase II clinical efficacy results were reviewed for whether they supported the likelihood of amyloid plaque reduction to predict clinical benefit, rather than whether they directly provided substantial evidence of effectiveness.

Drug Review Profile Drug Review

Amylyx’s Relyvrio: US FDA Review Shows Regulatory Flexibility Can Come In Many Forms

Pink Sheet Drug Review Profile looks at how the agency determined that Amylyx’s ‘conceptual basis’ for use of the drug’s two active ingredients to treat ALS was enough to satisfy the combination drug rule; the sponsor also was allowed to submit certain animal studies and drug-drug interaction data during the course of the NDA review.

Drug Review Profile Approvals

Early PHOENIX Data Would Not Have Aided Approval Decision On Amylyx’s Relyvrio – US FDA

Agency considered Amylyx’s plan to submit unblinded efficacy data from the ongoing Phase III trial during the ALS drug's NDA review but ultimately decided against it, citing concerns about study integrity and the small amount of data likely to be available.

Drug Review Profile Drug Review

Real-World Evidence: Amylyx’s Use Of External Controls To Frame Relyvrio’s Survival Benefit Rife With Problems

The post hoc comparison of an exploratory, long-term survival benefit in the CENTAUR trial to historical controls in two ALS patient databases lacked prespecification and a common treatment protocol, and potential differences in prognostic factors may have confounded the results.

Drug Review Profile Drug Review
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