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FDA Approves Retifanlimab Plus Carboplatin/Paclitaxel for Advanced Anal Cancer Following PODIUM-303 Trial Success

Drug Approval
  • The FDA approved retifanlimab in combination with carboplatin/paclitaxel for advanced anal cancer in May 2025, based on the PODIUM-303 study results.
  • The PODIUM-303 trial demonstrated improved progression-free survival (9.3 vs 7.4 months) and response rates (56% vs 44%) with the addition of retifanlimab to standard chemotherapy.
  • Current NCCN guidelines now include carboplatin/paclitaxel plus retifanlimab as a category 2B evidence-based approach for first-line treatment of advanced squamous cell anal cancer.
  • The InterAACT study previously established carboplatin/paclitaxel as standard care, showing similar response rates but improved survival and tolerability compared to cisplatin/5-FU.

Soleno Therapeutics Raises $200 Million to Fund Commercialization of First FDA-Approved Prader-Willi Syndrome Therapy

Drug ApprovalRare Disease
  • Soleno Therapeutics completed a $200 million public offering at $85 per share to fund commercialization of VYKAT XR, the first FDA-approved therapy for hyperphagia in Prader-Willi syndrome patients.
  • The company received FDA approval for VYKAT XR on March 26, 2025, marking a significant milestone for treating this rare genetic disorder.
  • Proceeds will also support regulatory and market development activities in the European Union and further research and development efforts.
  • The offering included 2,352,941 shares with underwriters holding a 30-day option to purchase an additional 352,941 shares at the same price.

FDA Approves First Year-Long Flea and Tick Injectable for Dogs

Drug Approval
  • The FDA has approved BRAVECTO QUANTUM, the first and only parasiticide that protects dogs against fleas and ticks for an entire year with a single injection.
  • The injectable suspension contains fluralaner and is indicated for dogs and puppies 6 months of age and older, with availability expected at veterinary clinics nationwide by August 2025.
  • BRAVECTO QUANTUM provides 12-month protection against most tick species and fleas, though protection against lone star ticks is limited to 8 months.
  • The product has already been approved in more than 50 countries worldwide, including Australia, New Zealand, and the European Union prior to U.S. approval.

Pelthos Therapeutics Launches ZELSUVMI, First FDA-Approved At-Home Treatment for Molluscum Contagiosum

Drug Approval
  • Pelthos Therapeutics has commercially launched ZELSUVMI (berdazimer) topical gel 10.3%, the first FDA-approved prescription medication for molluscum contagiosum that can be applied at home by patients or caregivers.
  • The nitric oxide-releasing gel demonstrated significant efficacy in the largest Phase 3 molluscum study to date, achieving complete lesion clearance in 33% of patients versus 19.7% with placebo at 12 weeks.
  • Molluscum contagiosum affects an estimated 16.7 million Americans with up to 6 million new cases annually, yet up to 73% of pediatric cases go untreated due to limited therapeutic options and inconvenient procedural treatments.

A Phase 3 Molluscum Contagiosum Efficacy and Safety Study

NCT04535531CompletedPhase 3
Novan, Inc.
Posted 9/1/2020

Jazz Pharmaceuticals Names Renee Gala as CEO in Strategic Leadership Transition

Drug Approval
  • Jazz Pharmaceuticals' Board of Directors unanimously selected Renee Gala as President and CEO, effective August 11, 2025, succeeding co-founder Bruce Cozadd who will remain as Board Chairperson.
  • Gala's leadership has driven Jazz's transformation with nearly 90% revenue growth and diversified portfolio expansion, positioning the company for sustainable growth with a robust oncology and neuroscience pipeline.
  • The leadership transition comes as Jazz pursues ambitious growth targets including a $5 billion revenue goal by 2025, with key regulatory milestones and clinical readouts expected in the coming months.

FDA Publishes Over 200 Drug Rejection Letters in Historic Transparency Initiative

Drug Approval
  • The FDA published more than 200 Complete Response Letters (CRLs) from 2020-2024 for drug applications that were initially rejected but later approved, marking a significant step toward increased regulatory transparency.
  • Commissioner Marty Makary stated that drug developers have been "playing a guessing game" with FDA navigation, emphasizing the need for greater predictability in the approval process.
  • The initiative addresses a longstanding issue where sponsors historically avoided mentioning 85% of FDA safety and efficacy concerns when publicly announcing rejections, according to a 2015 FDA analysis.
  • The letters are now accessible through openFDA, the first centralized database of past CRLs, with redactions for trade secrets and confidential commercial information.

FDA Accepts Taiho's Application for INQOVI-Venetoclax Combination in Acute Myeloid Leukemia

Drug Approval
  • The FDA has accepted Taiho Oncology's supplemental new drug application for INQOVI (decitabine and cedazuridine) plus venetoclax to treat adults with newly diagnosed acute myeloid leukemia who are ineligible for intensive induction chemotherapy.
  • The Phase 2b ASCERTAIN-V trial demonstrated a 46.5% complete response rate in 101 patients, with median overall survival estimated at 15.5 months and no new safety concerns reported.
  • If approved, this would represent the first all-oral combination treatment option for AML patients ineligible for intensive chemotherapy, with a PDUFA target action date of February 25, 2026.

Johnson & Johnson Seeks European Approval to Expand AKEEGA for Metastatic Prostate Cancer with Genetic Alterations

Drug ApprovalPrecision Medicine
  • Janssen-Cilag applied to the European Medicines Agency to expand AKEEGA usage for adults with metastatic hormone-sensitive prostate cancer who have homologous recombination repair gene alterations.
  • Over 20% of mHSPC patients have HRR gene alterations, including BRCA1/2 genes, and these patients often experience worse outcomes with limited treatment options.
  • The application is supported by Phase 3 AMPLITUDE study results showing the niraparib and abiraterone acetate combination significantly delayed cancer progression and symptom worsening.
  • AKEEGA combines niraparib and abiraterone acetate, targeting a specific patient population with genetic changes that affect DNA repair mechanisms.

Merck Announces $10 Billion Acquisition of Verona Pharma to Expand COPD Treatment Portfolio

Drug Approval
  • Merck has entered into a definitive agreement to acquire Verona Pharma for $10 billion, adding the first-in-class COPD treatment Ohtuvayre to its respiratory portfolio.
  • Ohtuvayre represents the first novel inhaled mechanism for COPD maintenance treatment in over 20 years, combining bronchodilator and anti-inflammatory effects in a single molecule.
  • The acquisition addresses Merck's need to diversify revenue streams ahead of Keytruda's patent expiry in 2028, with the transaction expected to close in Q4 2025.
  • Ohtuvayre generated over 96% of Verona's $76 million first-quarter revenue and is also being evaluated for non-cystic fibrosis bronchiectasis treatment.

Johnson & Johnson Submits FDA Application to Expand Caplyta for Schizophrenia Relapse Prevention

Drug Approval
  • Johnson & Johnson has submitted a supplemental New Drug Application to the FDA seeking expanded approval for Caplyta (lumateperone) to prevent schizophrenia relapse.
  • The antipsychotic drug is already FDA-approved for treating schizophrenia, and this expansion could significantly strengthen J&J's mental health portfolio.
  • Analysts estimate Caplyta has potential to achieve over $5 billion in peak sales, supporting the company's strategy to bolster revenue through innovative medicines.
  • The expanded indication aligns with J&J's efforts to offset challenges from patent expirations, including the loss of exclusivity for STELARA.
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