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FDA Grants RMAT Designation to Grace Science's Gene Therapy for NGLY1 Deficiency

FDA Grants RMAT Designation to Grace Science's Gene Therapy for NGLY1 Deficiency

FDA Grants RMAT Designation to Grace Science's Gene Therapy for NGLY1 Deficiency
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FDA Approves Travere's Filspari as First Treatment for Rare Kidney Disease FSGS

FDA Approves Travere's Filspari as First Treatment for Rare Kidney Disease FSGS

FDA Approves Travere's Filspari as First Treatment for Rare Kidney Disease FSGS
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FDA Grants RMAT Designation to Grace Science's GS-100 Gene Therapy for NGLY1 Deficiency

FDA Grants RMAT Designation to Grace Science's GS-100 Gene Therapy for NGLY1 Deficiency

The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to GS-100, a gene therapy developed by Grace Science, LLC, for treating NGLY1 Deficiency. This ultra-rare genetic disease affects children and currently has no approved therapies. GS-100 is an investigational adeno-associated viral vector therapy designed to replace the deficient NGLY1 protein. The RMAT designation is based on promising early clinical evidence showing improvements in motor function and cognitive skills in patients treated with GS-100. This designation aims to expedite the development and review process, potentially leading to accelerated approval.
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PolyPid Presents Phase 3 Data on D-PLEX100's Sustained Antibiotic Release for Surgical Site Infections

PolyPid Presents Phase 3 Data on D-PLEX100's Sustained Antibiotic Release for Surgical Site Infections

PolyPid Presents Phase 3 Data on D-PLEX100's Sustained Antibiotic Release for Surgical Site Infections
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Antiva Biosciences Reports Positive Results from ABI-2280 Trial for Cervical HPV Treatment

Antiva Biosciences Reports Positive Results from ABI-2280 Trial for Cervical HPV Treatment

Antiva Biosciences has announced promising results from its Phase 1b/2 clinical trial of ABI-2280, a novel topical therapeutic for treating persistent high-risk cervical human papillomavirus (hrHPV) infections. The trial, presented at the Society of Gynecologic Oncology Annual Meeting, involved 139 women aged 25 to 55 with documented hrHPV infections. The study achieved its primary and secondary endpoints, showing statistically significant improvements in hrHPV negativity at weeks 12 and 24 compared to placebo. Notably, 78% of patients with the HPV16 genotype achieved negativity at week 12, compared to 0% in the placebo group. The treatment was well tolerated, with mild to moderate adverse events localized to the treatment area.
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FDA's Second Rejection of Replimune's Melanoma Drug RP1 Sparks Strategic Overhaul

FDA's Second Rejection of Replimune's Melanoma Drug RP1 Sparks Strategic Overhaul

FDA's Second Rejection of Replimune's Melanoma Drug RP1 Sparks Strategic Overhaul
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FDA Grants Priority Review to Ifinatamab Deruxtecan for Small Cell Lung Cancer Treatment

FDA Grants Priority Review to Ifinatamab Deruxtecan for Small Cell Lung Cancer Treatment

The U.S. Food and Drug Administration (FDA) has granted Priority Review to the Biologics License Application (BLA) for ifinatamab deruxtecan, a potential first-in-class B7-H3 directed DXd antibody drug conjugate developed by Daiichi Sankyo and Merck. This investigational drug is intended for adult patients with extensive-stage small cell lung cancer (ES-SCLC) who have experienced disease progression following platinum-based chemotherapy. The Priority Review status indicates that the FDA sees significant potential for the drug to improve treatment outcomes compared to existing options. The application is also being reviewed under the Real-Time Oncology Review (RTOR) program and Project Orbis, which aim to expedite the availability of effective cancer treatments. The Prescription Drug User Fee Act (PDUFA) date for the FDA's decision is set for October 10, 2026.
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FDA Grants Full Approval to Travere Therapeutics' Filspari for Rare Kidney Disease FSGS

FDA Grants Full Approval to Travere Therapeutics' Filspari for Rare Kidney Disease FSGS

FDA Grants Full Approval to Travere Therapeutics' Filspari for Rare Kidney Disease FSGS
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Telix Pharmaceuticals Advances in Radiopharmaceuticals with FDA Nod for Brain Cancer Imaging Agent

Telix Pharmaceuticals Advances in Radiopharmaceuticals with FDA Nod for Brain Cancer Imaging Agent

Telix Pharmaceuticals Advances in Radiopharmaceuticals with FDA Nod for Brain Cancer Imaging Agent
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Replimune Faces Challenges After Second FDA Rejection of Melanoma Drug

Replimune Faces Challenges After Second FDA Rejection of Melanoma Drug

Replimune Faces Challenges After Second FDA Rejection of Melanoma Drug
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FDA Rejects Replimune's Melanoma Therapy for Second Time

FDA Rejects Replimune's Melanoma Therapy for Second Time

FDA Rejects Replimune's Melanoma Therapy for Second Time
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Replimune Faces Uncertainty After FDA Rejects Melanoma Drug

Replimune Faces Uncertainty After FDA Rejects Melanoma Drug

Replimune Faces Uncertainty After FDA Rejects Melanoma Drug