FDA Accepts Sparsentan sNDA for Full Approval in Focal Segmental Glomerulosclerosis
• The FDA has accepted Travere Therapeutics' supplemental New Drug Application for traditional approval of sparsentan (Filspari) in focal segmental glomerulosclerosis, with a decision expected by January 2026.
• An FDA advisory committee meeting will be convened to review the application data, signaling the regulatory importance of this potential treatment option for FSGS patients.
• If approved, sparsentan would strengthen treatment options for FSGS, a rare kidney disorder with limited therapeutic alternatives and significant unmet medical needs.
Regeneron Wins $407 Million in Landmark Antitrust Case Against Amgen Over PCSK9 Drug Market
• A federal jury awarded Regeneron $407 million in damages after finding Amgen violated antitrust laws by using cross-therapeutic bundled rebates to block Praluent from competing with Repatha in the PCSK9 inhibitor market.
• The verdict includes $135.6 million in compensatory damages and $271.2 million in punitive damages, signaling a strong deterrent against anticompetitive bundling practices in pharmaceutical markets.
• Amgen was found to have leveraged its blockbuster drugs Enbrel and Otezla to force pharmacy benefit managers to exclude Praluent, creating an uneven playing field not based on clinical merit or price.
Leap Therapeutics Halves Workforce and Refocuses Cancer Drug Development Amid Market Challenges
• Leap Therapeutics has announced a significant restructuring, reducing its workforce by approximately 50% and narrowing the development focus of its lead cancer drug candidate in response to challenging market conditions.
• The strategic pivot aims to extend the company's cash runway while concentrating resources on the most promising clinical applications of its lead oncology asset, potentially improving its chances for regulatory success.
• This move follows similar restructuring trends across the biotech sector, with companies like Arcturus, NGM Bio, and Erasca all recently announcing staff reductions and pipeline reprioritizations to navigate the difficult funding environment.
Verrica Pharmaceuticals Reports Record Growth in YCANTH® Dispensed Units for Molluscum Contagiosum Treatment
• Verrica Pharmaceuticals achieved a milestone with over 10,000 YCANTH® dispensed applicator units in Q1 2025, representing a 16.7% sequential increase from the previous quarter.
• The company reports normalized distribution inventory levels, indicating that dispensed units will now more closely track actual demand and gross revenue.
• Following a commercial restructuring, Verrica is positioned for sustainable revenue growth as it works to establish YCANTH® as the standard of care for molluscum contagiosum.
FDA Approves First Treatment for Cerebrotendinous Xanthomatosis: Mirum's Chenodiol Tablets
• The FDA has granted historic approval to chenodiol tablets (Ctexli) as the first-ever treatment for cerebrotendinous xanthomatosis, a rare genetic lipid storage disease affecting multiple organ systems.
• Phase 3 RESTORE study demonstrated chenodiol's efficacy with a statistically significant 20-fold reduction in bile alcohols compared to placebo (P<0.0001) in CTX patients.
• The treatment, administered as 250mg tablets three times daily, comes with monitoring requirements for liver toxicity and showed common side effects including diarrhea and headache.
Scotiabank Raises Travere Therapeutics' Price Target Following Planned Filspari sNDA Filing
• Scotiabank analyst Greg Harrison has increased Travere Therapeutics' price target to $32 from $27, maintaining an Outperform rating on the company's shares.
• Travere Therapeutics has announced plans to submit a supplemental New Drug Application (sNDA) for Filspari, signaling potential positive discussions with the FDA.
• The analyst's confidence suggests that preliminary FDA discussions were favorable, though final approval remains subject to regulatory review.
Travere Therapeutics Plans sNDA Submission for FILSPARI to Treat Rare Kidney Disease FSGS
• Travere Therapeutics has completed a Type C meeting with the FDA and plans to submit a supplemental New Drug Application for FILSPARI to treat focal segmental glomerulosclerosis by Q1 2025.
• If approved, FILSPARI would become the first FDA-approved treatment for FSGS, a rare kidney disorder affecting over 40,000 patients in the U.S. that can lead to kidney failure.
• The sNDA submission will be based on data from the Phase 3 DUPLEX and Phase 2 DUET studies, supported by recent PARASOL workgroup findings linking proteinuria reduction to improved kidney outcomes.
Evorpacept Shows Promise in HER2-Positive Gastric Cancer: Updated ASPEN-06 Data
• ALX Oncology's ASPEN-06 Phase 2 trial evaluates evorpacept, a CD47-blocker, in HER2-positive advanced gastric cancer patients who have been previously treated.
• Updated data from the ASPEN-06 trial were presented at the 2025 ASCO Gastrointestinal Cancers Symposium (ASCO GI).
• The virtual event hosted by ALX Oncology on January 23, reviewed the ASPEN-06 data, focusing on evorpacept's potential in immuno-oncology.
• The ASPEN-06 trial is a phase 2/3 study of evorpacept in patients with HER2-overexpressing gastric/gastroesophageal cancer.
Krystal Biotech and CRISPR Therapeutics Announce Key Updates in Gene Therapy Programs
• Krystal Biotech reported a 473% increase in full-year revenue, driven by VYJUVEK sales, and anticipates European and Japanese regulatory decisions in 2025.
• Krystal Biotech's KB407 for cystic fibrosis received full sanctioning for its Phase 1 CORAL-1 study, with interim molecular data expected mid-2025.
• CRISPR Therapeutics reported positive momentum in the CASGEVY launch, with over 50 activated treatment centers and patients initiating cell collection globally.
• CRISPR Therapeutics expects key clinical data updates in 2025 for CTX112 in oncology and autoimmune diseases, and for CTX310 and CTX320 in cardiovascular programs.
EMA Panel Recommends Sparsentan for IgA Nephropathy, Paving Way for EU Approval
• The EMA's human medicines committee (CHMP) has recommended sparsentan for IgA nephropathy (IgAN) treatment in adults with significant proteinuria.
• Sparsentan, developed by Travere Therapeutics and CSL Vifor, is a dual endothelin type A (ETA) and angiotensin II subtype 1 (AT1) receptor antagonist.
• The positive opinion puts sparsentan on track to potentially become the first-in-class therapy for IgAN in Europe, following accelerated approval in the US as Filspari.
• Clinical data from the PROTECT study supported the recommendation, demonstrating a reduction in proteinuria, though eGFR improvement was not significant.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
LIB Therapeutics' Lerodalcibep BLA Accepted by FDA for LDL-C Reduction
• The FDA has accepted LIB Therapeutics' BLA for lerodalcibep, a PCSK9 inhibitor, with a PDUFA date set for December 12, 2025.
• Lerodalcibep targets LDL-C reduction in patients with ASCVD or at high risk, including those with heterozygous and homozygous familial hypercholesterolemia.
• Clinical trials demonstrated robust LDL-C lowering with convenient monthly subcutaneous injection and no need for refrigeration, enhancing patient adherence.
• LIB Therapeutics anticipates submitting a Marketing Authorization Application to the EMA in Q2 2025, with commercial launch preparations underway.
FDA Approves Ozempic for Reducing Kidney Disease Risks in Type 2 Diabetes Patients
• The FDA has approved Ozempic to reduce the risk of kidney disease worsening, kidney failure, and cardiovascular death in adults with type 2 diabetes and chronic kidney disease.
• A Phase 3 clinical trial demonstrated a 24% reduction in the risk of kidney disease progression and cardiovascular death compared to placebo with Ozempic.
• Ozempic is now the most broadly indicated GLP-1 receptor agonist, offering a new treatment option for millions of adults with type 2 diabetes and chronic kidney disease.
• The approval was based on the FLOW trial, which showed significant benefits in kidney and cardiovascular outcomes, leading to early termination due to efficacy.
Novartis and Apellis Advance C3G Therapies Towards FDA Submission
• Novartis' Fabhalta (iptacopan) demonstrated significant proteinuria reduction in the Phase III APPEAR-C3G trial, meeting its primary endpoint and supporting regulatory submissions.
• Apellis' Empaveli (pegcetacoplan) showed a substantial reduction in proteinuria and C3c deposit clearance in the Phase III VALIANT trial, indicating early efficacy in C3G patients.
• Nephrologists express a strong interest in new C3G therapies that can slow eGFR decline and reduce proteinuria, addressing the urgent need for innovative treatments.
• Spherix data suggests treatment preferences may be influenced by administration route, with Fabhalta's oral administration potentially offering an advantage.
Industry Leaders Unite to Tackle Patient Recruitment Challenges in Rare Disease Clinical Trials
• Over 80% of U.S. clinical trials fail to meet patient enrollment timelines, highlighting the significant challenges in rare disease research and patient recruitment.
• Leading pharmaceutical companies including Biogen, Astellas Pharma, and Novo Nordisk are collaborating to address patient recruitment obstacles through innovative strategies and enhanced diversity initiatives.
• The Patient Recruitment for Rare Disease Trials Summit brings together experts to explore solutions for patient engagement, data sharing, and fair compensation while maintaining trial integrity.
FDA Grants Full Approval to Travere's Filspari for IgA Nephropathy
• The FDA granted full approval to Travere Therapeutics' Filspari (sparsentan) for IgA nephropathy, allowing broader use for patients at risk of disease progression.
• The approval was based on the PROTECT study, which demonstrated Filspari significantly slowed kidney function decline over two years compared to irbesartan.
• Filspari is now positioned as a foundational, non-immunosuppressive treatment option, potentially replacing the current standard of care for IgAN patients.
• The label update removes a specific urine protein level requirement, expanding the eligible patient population and increasing Filspari's market potential.
14 Breakthrough Drugs Poised for Blockbuster Status by 2027: Clarivate Analysis
• Immunological and inflammatory disease treatments dominate the list of potential blockbuster drugs, with targeted biomarker approaches leading the innovation landscape.
• Novel therapies include first-in-class treatments like bimekizumab for psoriasis, lecanemab for Alzheimer's, and teplizumab for type 1 diabetes, each offering significant advances in their respective fields.
• Several candidates, including Gilead's lenacapavir for HIV and BioMarin's Roctavian for hemophilia A, represent transformative approaches with potential to revolutionize treatment paradigms through innovative delivery methods.
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