July FDA Review Calendar: GSK, Roche, Regeneron Face Pivotal Decisions on Oncology, Rare Disease, and Endocrine Therapies

In late July, the U.S. FDA will face a wave of intensive new drug reviews, focusing on popular fields such as hematological tumors, rare diseases, and endocrinology, involving pharmaceutical companies such as Roche, GSK, and Regeneron. The review content covers the expansion of indications for targeted therapies.Star ADC Drug Expected to Return to the Market, as well as a promising new drug with the potential to reshape the landscape of pediatric and rare disease treatment.

Roche's CD20×CD3 Bispecific Antibody Expands Indications
Roche's submitted supplemental Biologics License Application (sBLA) aims to combine its CD20×CD3 bispecific antibody Columvi with chemotherapy for patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who have received at least one prior line of therapy. Currently, Columvi is only approved for patients who have undergone at least two prior lines of therapy. This sBLA submission is based on the STARGLO Phase III data, which demonstrated that the combination with chemotherapy reduced the risk of death by 41%. If approved, it could become a standard treatment option after first-line therapy, with the FDA expected to make a decision by July 20.

Replimune Submits RP1 in Combination with Opdivo for Advanced Melanoma
Replimune's RP1 (an oncolytic immunotherapy based on modified herpes simplex virus) in combination with BMS PD-1 inhibitor Opdivo for advanced melanoma patients who failed anti-PD-1 treatment is expected to release results on July 22.According to the IGNYTE study, the ORR of the RP1 combination regimen reached 32.9%, with a complete response rate of 15% and a 3-year overall survival rate exceeding 54%.

GSK ADC Drug BlenrepMake a Comeback”
Following the 2022 withdrawal of Blenrep's marketing application due to the failure of the DREAMM-3 trial, GSK has reapplied for the use of Blenrep in relapsed/refractory multiple myeloma, supported by positive data from two Phase III studies, DREAMM-7 and DREAMM-8. Earlier interim analyses showed clinically meaningful improvements in both PFS and OS.FDA to Make Decision on July 23

Sobi Expands Doptelet Indication to Pediatric ITP
Oral TPO Agonist Doptelet Poised for Approval in Children Aged 1 Year and Older with Immune Thrombocytopenia (ITP). Data from the AVA-PED-301 trial demonstrated sustained platelet response, with a target review date of July 24.

Skytrofa Challenges the Adult GHD Market for the First Time
Ascendis Pharma's long-acting growth hormone, Skytrofa, is currently used for the treatment of pediatric GHD and is now seeking an indication expansion for adult growth hormone deficiency. Based on the foresiGHt Phase III study data, Skytrofa significantly reduces abdominal fat and increases lean body mass, with the review results to be announced by July 27.

Apellis Advances Empaveli for Two Rare Kidney Diseases
In the rare disease field, Apellis' Empaveli is proposed to expand to two rare kidney diseases — C3G and IC-MPGN. In the VALIANT Phase III study, the drug significantly reduced proteinuria by 68% and decreased C3c deposition within 26 weeks, potentially offering the first targeted complement treatment for rare nephritis. The review result is expected on July 28.。

PTC Rare Disease Drug Sepiapterin Challenges PKU Treatment Landscape
For Phenylketonuria (PKU), the oral drug Sepiapterin, applied for by PTC, is expected to receive approval on July 29. The drug can activate and stabilize the PAH enzyme, reducing Phe levels by 63% in the APHENITY study, with an 85% treatment response rate, potentially rewriting the treatment paradigm for this rare metabolic disorder.

Regeneron Revisits FL Bispecific Antibody Therapy Approval
Regeneron's CD20×CD3 bispecific antibody odronextamab, intended for relapsed/refractory follicular lymphoma (FL) as a treatment for patients failing two prior lines of therapy, was previously rejected by the FDA due to insufficient enrollment in the confirmatory trial. The OLYMPIA-1 trial has now completed enrollment. Data from the ELM-1 and ELM-2 studies show an ORR of 80% and a CR rate of 74%. The FDA will issue its decision on July 30.

Summary：
From hematologic tumors to multiple myeloma, from rare pediatric diseases to metabolic disorder treatments, a series of FDA approvals in late July will profoundly impact the global pharmaceutical landscape.This is not only a window period for the implementation of innovative achievements, but also marks the entry of Biotech into a new round of competition and reshuffle.

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