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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.

Clinical Phase III Trial Treosulfan-based Conditioning Versus Reduced-intensity Conditioning (RIC)

NCT00822393CompletedPhase 3
medac GmbH
Posted 11/24/2008

A Study to Compare Tivozanib (AV-951) to Sorafenib in Subjects With Advanced Renal Cell Carcinoma

NCT01030783CompletedPhase 3
AVEO Pharmaceuticals, Inc.
Posted 12/1/2009

A Study to Compare Tivozanib Hydrochloride to Sorafenib in Participants With Refractory Advanced Renal Cell Carcinoma (RCC)

NCT02627963CompletedPhase 3
AVEO Pharmaceuticals, Inc.
Posted 5/24/2016

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.

Fziomed Receives FDA De Novo Authorization for Oxiplex Gel in Lumbar Spine Surgery

Drug Approval
  • Fziomed has received FDA De Novo classification and marketing authorization for Oxiplex gel, making it the first and only FDA-authorized intraoperative gel specifically indicated to reduce postoperative leg pain and neurological symptoms in adult patients undergoing lumbar spine procedures.
  • The approval was supported by Level 1 clinical evidence from multiple U.S. randomized clinical trials and international post-marketing studies, demonstrating Oxiplex's strong safety profile and clinical benefit after over 20 years of real-world use outside the United States.
  • Oxiplex gel has been used in more than 750,000 spine procedures worldwide since 2002 and has the potential to become a new standard of care as an adjunctive therapy for addressing significant unmet clinical needs in spine surgery.

Insightec Receives FDA Approval for Bilateral Focused Ultrasound Treatment in Advanced Parkinson's Disease

Drug Approval
  • The FDA has approved Insightec's Exablate Neuro platform for staged bilateral pallidothalamic tractotomy treatment in patients with advanced Parkinson's disease, expanding therapeutic options for over 50,000 patients.
  • The incisionless procedure uses MRI-guided focused ultrasound to precisely target brain areas without requiring implanted hardware, offering a lower risk profile compared to traditional surgical interventions.
  • Clinical trials conducted at nine centers across the U.S., Europe, and Asia demonstrated encouraging outcomes supporting FDA approval, with full results expected to be published later this year.
  • Insightec plans a limited launch of the bilateral procedure in select centers during 2025, with ongoing efforts to establish routine reimbursement pathways for broader patient access.

Evaluation of the Safety and Effectiveness of Bilateral Exablate Ablation of the Pallidothalamic Tract (PTT) for the Treatment of the Motor Complications of Parkinson's Disease (PD)

NCT04728295Active, Not RecruitingNot Applicable
InSightec
Posted 5/14/2021

Johnson & Johnson Submits FDA Application for CAPLYTA Schizophrenia Relapse Prevention Based on 63% Risk Reduction Data

Drug Approval
  • Johnson & Johnson submitted a supplemental New Drug Application to the FDA for CAPLYTA (lumateperone) for schizophrenia relapse prevention based on Phase 3 data showing 63% reduction in relapse risk versus placebo.
  • The multicenter, double-blind trial demonstrated significantly longer time to relapse during 26-week treatment phase (p=0.0002) and delayed time to all-cause discontinuation (p=0.0007).
  • CAPLYTA is already FDA-approved for schizophrenia treatment and bipolar depression, with this expansion potentially addressing a critical unmet need in long-term schizophrenia management.
  • The safety profile remained consistent with existing clinical data, with headache being the most commonly reported adverse event at rates exceeding placebo.

Novartis Receives Approval for First Malaria Treatment Designed for Newborns and Young Infants

Drug ApprovalDrug Approval
  • Novartis has received Swiss regulatory approval for Coartem Baby, the first malaria treatment specifically formulated for newborns and infants weighing 2-5 kilograms.
  • The approval addresses a critical treatment gap, as existing malaria drugs were only tested in children aged 6 months and older, creating dosing risks for babies with immature liver function.
  • The new treatment will be rolled out in African countries within weeks on a largely not-for-profit basis, targeting regions where malaria caused 597,000 deaths in 2023.
  • Eight African nations participated in the approval process and are expected to be among the first to access the medicine, which is dissolvable in breast milk and has a sweet cherry flavor.

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