Prescription Drug User Fee Act

Checkpoint Therapeutics Announces Cosibelimab Longer-Term Results Demonstrating Substantial Increases in Complete Response Rates in Advanced Cutaneous Squamous Cell Carcinoma

Retrieved on: 
Thursday, July 27, 2023
Radiation, Neoplasm, Immunotherapy, Autoimmunity, Prescription Drug User Fee Act, Ira Pastan, Patient, Autoimmune disease, PD-1, Oncology, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, PD-L1, FC, Food, Safety, Pharmaceutical industry

WALTHAM, Mass., July 27, 2023 (GLOBE NEWSWIRE) -- Checkpoint Therapeutics, Inc. (“Checkpoint”) (Nasdaq: CKPT), a clinical-stage immunotherapy and targeted oncology company, today announced new, longer-term data for cosibelimab from its pivotal studies in locally advanced and metastatic cutaneous squamous cell carcinoma (“cSCC”). These results demonstrate a deepening of response over time, resulting in substantially higher complete response rates than previously reported. Furthermore, responses continue to remain durable over time with the median duration of response not yet reached in either group. Results determined by independent central review by treatment group were as follows:

Key Points: 
  • “We are excited to see the substantial increases in the rate of patients experiencing a complete response of their cSCC tumors with further cosibelimab treatment in both our locally advanced and metastatic pivotal trials,” said James Oliviero, President and Chief Executive Officer of Checkpoint.
  • Mr. Oliviero continued, “Unlike PD-1 inhibitors, cosibelimab does not interrupt the body’s PD-1/PD-L2 pathway, which we believe results in cosibelimab’s low rates of autoimmunity.
  • The application is filed and under review with a Prescription Drug User Fee Act (“PDUFA”) goal date of January 3, 2024.
  • Checkpoint plans to present these updated results at an upcoming medical conference.

Ipsen delivers solid H1 2023 results and upgrades its full-year guidance

Retrieved on: 
Thursday, July 27, 2023
IPN, Heart, Cholestatic pruritus, Maintenance, CHC, FY, Ascending cholangitis, Civil service commission, ADR, Prescription Drug User Fee Act, Fibrodysplasia ossificans progressiva, Ursodeoxycholic acid, Account, Ipsen, Galderma, Bank statement, Oxaliplatin, COMP, Rare disease, New Drug Application, European Directive on Traditional Herbal Medicinal Products, Research, Committee, EMA, ICC, Primary biliary cholangitis, Report, FOP, Patient, Committee for Medicinal Products for Human Use, PDUFA, Webcast, Consumer, Growth, Ageing, Alagille syndrome, Indoor Living, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Food, European Commission, Safety, CHMP, Pharmaceutical industry, Medical device, Cryptocurrency, ALGS, Oncology, Neuroscience, Odevixibat, Euronext

The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.

Key Points: 
  • The performance in the first half of the year is reflected in today’s upgrade to our full-year guidance.
  • The Company’s auditors performed a limited review of the H1 2023 condensed consolidated financial statements.
  • Ipsen intends to publish its year-to-date and third-quarter sales update on 26 October 2023.
  • The performance shown in this announcement covers the six-month period to 30 June 2023 (H1 2023) and the three-month period to 30 June 2023 (Q2 2023), compared to six-month period to 30 June 2022 (H1 2022) and the three-month period to 30 June 2022 (Q2 2022), respectively, unless stated otherwise.

Elevar Therapeutics Announces Publication of Phase 3 CARES 310 Study Results in The Lancet

Retrieved on: 
Tuesday, July 25, 2023
Camrelizumab, ACC, Hepatocellular carcinoma, Progression-free survival, Food, TKI, Adenoid cystic carcinoma, Prescription Drug User Fee Act, Patient, Sorafenib, PDUFA, PD-1, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, HCC, Pharmaceutical industry, Lancet

FORT LEE, N.J., July 24, 2023 (GLOBE NEWSWIRE) -- Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced the publication of its Phase 3 CARES 310 study results in The Lancet.

Key Points: 
  • FORT LEE, N.J., July 24, 2023 (GLOBE NEWSWIRE) -- Elevar Therapeutics, Inc., a majority-owned subsidiary of HLB Co., Ltd. and a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced the publication of its Phase 3 CARES 310 study results in The Lancet.
  • The study assessed the combination of Elevar’s drug candidate rivoceranib, an oral TKI, in combination with camrelizumab, a PD-1 inhibitor, as a first-line therapy for unresectable hepatocellular carcinoma (uHCC).
  • “As evidenced in the CARES 310 study, camrelizumab plus rivoceranib demonstrate significant promise as a potentially improved therapy for advanced hepatocellular carcinoma,” said Saeho Chong, Elevar chief executive officer.
  • With efficacy results generally consistent across all subgroups, the CARES 310 data suggested the combination confers a benefit in a global uHCC population.

BeiGene Announces FDA Acceptance of sNDA for Fifth BRUKINSA® Indication

Retrieved on: 
Wednesday, July 12, 2023
Biotechnology, FDA, Pharmaceutical, Health, Obinutuzumab, Prescription Drug User Fee Act, BGNE, Arm, Diarrhea, Follicular lymphoma, DOR, Safety, Initiative, Multimedia, SSE, Shanda, HIV disease progression rates, Patient, Risk, European, United Kingdom, Fever, Death, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, ORR, Food, Fatigue, Pharmaceutical industry, Medical device, Hematology, BeiGene

BRUKINSA was previously granted Fast Track and Orphan designation for this indication.

Key Points: 
  • BRUKINSA was previously granted Fast Track and Orphan designation for this indication.
  • The FDA has assigned a target action date in the first quarter of 2024, under the Prescription Drug User Fee Act.
  • Safety results from the ROSEWOOD study were consistent with previous studies of both medicines.
  • The global BRUKINSA development program includes more than 4,900 subjects enrolled to-date in 29 countries and regions.

Iovance Biotherapeutics Announces Regulatory and Clinical Updates for TIL Therapy in Advanced Non-Small Cell Lung Cancer

Retrieved on: 
Monday, July 10, 2023
IOV, Prescription Drug User Fee Act, TIL, DOR, RECIST, Cancer, NSCLC, ROS, Patient, SAN, EGFR, Interleukin, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, ORR, Food, Disease, ALK, Pharmaceutical industry, Iovance Biotherapeutics

SAN CARLOS, Calif., July 10, 2023 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a biotechnology company focused on innovating, developing, and delivering novel polyclonal tumor infiltrating lymphocyte (TIL) therapies for patients with cancer, today announced positive regulatory and clinical updates related to its registrational single-arm Phase 2 IOV-LUN-202 trial in post-anti-PD-1 NSCLC.

Key Points: 
  • Based on this FDA feedback, Iovance completed a preliminary analysis of the IOV-LUN-202 trial.
  • Based on the regulatory discussions, Iovance plans to enroll a total of approximately 120 patients into the registrational IOV-LUN-202 trial.
  • As previously announced , Iovance is also preparing to meet with the FDA this year to discuss a randomized confirmatory trial of LN-145 in frontline advanced NSCLC patients.
  • This confirmatory trial in frontline advanced NSCLC is expected to be well underway at the time of a potential approval in advanced post-anti-PD-1 NSCLC.

Alnylam Announces Date of Planned FDA Advisory Committee Meeting for ONPATTRO® (patisiran) for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Retrieved on: 
Friday, June 30, 2023
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Prescription Drug User Fee Act, Nyctalopia, Therapy, Register, Premedication, Serum, New Drug Application, Vitamin A deficiency, Vitamin A, Federal, IRR, Abdominal pain, Medication, Amyloidosis, Back pain, ATTR, Patient, Risk, Paracetamol, Circulatory system, Nausea, Periodic breathing, RNA interference, Cardiomyopathy, Polyneuropathy, Headache, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, RDA, Food, Dietary supplement

As previously announced, the FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act.

Key Points: 
  • As previously announced, the FDA has set an action date of October 8, 2023 under the Prescription Drug User Fee Act.
  • An advanced display of the public notice is available in the Federal Register here .
  • Patisiran is the established name for ONPATTRO®, which is currently approved by the U.S. FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
  • ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

bluebird bio Announces FDA Priority Review of the Biologics License Application for lovotibeglogene autotemcel (lovo-cel) for Patients with Sickle Cell Disease (SCD) 12 years and Older with a History of Vaso-Occlusive Events

Retrieved on: 
Wednesday, June 21, 2023
Biotechnology, FDA, Health, Genetics, Pharmaceutical, Biologics license application, Priority review, File, SCD, Ronas Voe, History, Regenerative medicine, Prescription Drug User Fee Act, Patient, PDUFA, Sickle cell disease, Diagnosis, Caregiver, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, BLA, Food, DSM-IV codes, Safety, Disease, Pharmaceutical industry

bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review.

Key Points: 
  • bluebird bio, Inc. (Nasdaq: BLUE) today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for lovotibeglogene autotemcel (lovo-cel) for priority review.
  • Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events (VOEs).
  • The agency has set a Prescription Drug User Fee Act (PDUFA) goal date of December 20, 2023.
  • The FDA previously granted lovo-cel orphan drug designation, fast track designation, regenerative medicine advanced therapy (RMAT) designation, and rare pediatric disease designation.

ARS Pharmaceuticals Announces PDUFA Date Extension for neffy® (Intranasal (IN) Epinephrine) for the Treatment of Allergic Reactions (Type 1), Including Anaphylaxis

Retrieved on: 
Tuesday, June 20, 2023
Prescription Drug User Fee Act, Allergy, ARS, Anxiety, European Medicines Agency, New Drug Application, Adrenaline, NDA, Patient, Anaphylaxis, SAN, PDUFA, Nasal administration, Entrepreneurship, FDA, Regulation of tobacco by the U.S. Food and Drug Administration, Food, Caregiver, Pharmaceutical industry, Agency

SAN DIEGO, June 20, 2023 (GLOBE NEWSWIRE) -- ARS Pharmaceuticals, Inc .

Key Points: 
  • SAN DIEGO, June 20, 2023 (GLOBE NEWSWIRE) -- ARS Pharmaceuticals, Inc .
  • The updated PDUFA target action date is September 19, 2023.
  • The additional time is needed for labeling and post-marketing commitment discussions regarding neffy, and ARS has submitted proposals for post-marketing commitments based on input from the PADAC meeting in May.
  • “FDA is working on labeling and post-marketing commitments as the final steps in the review process,” said Richard Lowenthal, Co-Founder, President and Chief Executive Officer, ARS Pharmaceuticals.

Catalyst Pharmaceuticals to License North American Rights to Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals

Retrieved on: 
Tuesday, June 20, 2023
Neuromuscular disease, LLP, Partnership, Catalyst Pharmaceuticals, New Drug Application, Calendar, NDA, DSM-IV codes, Duchenne, Duchenne muscular dystrophy, Cooley LLP, Prescription Drug User Fee Act, DMD, Patient, PDUFA date, PDUFA, Rare, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, CHF, Treatment, Food, Pharmaceutical industry, Vamorolone, Catalysis

Vamorolone is Currently Under Review with the FDA (PDUFA Date of October 26, 2023) for the Treatment of Duchenne Muscular Dystrophy, a Rare Neuromuscular Disease

Key Points: 
  • Vamorolone is Currently Under Review with the FDA (PDUFA Date of October 26, 2023) for the Treatment of Duchenne Muscular Dystrophy, a Rare Neuromuscular Disease
    Catalyst Expects to Launch Vamorolone in the U.S.
  • In addition to the North American rights, which consist of the United States, Canada, and Mexico territories, the transaction provides Catalyst the right of first negotiation for vamorolone in Europe and Japan should Santhera pursue partnership opportunities.
  • Furthermore, Catalyst and Santhera will enter into a Joint Steering Committee to oversee the development of vamorolone for additional indications beyond DMD.
  • Moelis & Company LLC is acting as the exclusive financial advisor to Catalyst, and Akerman LLP and Cooley LLP are acting as legal advisors to Catalyst.

Santhera Grants Exclusive North America License for Vamorolone to Catalyst Pharmaceuticals in Deal Valued at up to USD 231 Million Plus Royalties

Retrieved on: 
Tuesday, June 20, 2023
Centerview Partners, Treasury, Vamorolone, Catalyst Pharmaceuticals, Prescription Drug User Fee Act, JSC, Duchenne muscular dystrophy, Civil service commission, Marketing, Health care, European Medicines Agency, LR, NDA, European Directive on Traditional Herbal Medicinal Products, Medication, USD, Committee, EMA, U.S. FDA, Standstill, Patient, CEST, PDUFA, Committee for Medicinal Products for Human Use, MAA, Rare, FDA, EC, News, Muscular dystrophy, European Commission, MHRA, CHMP, BST, Tutoring, Pharmaceutical industry, Dentistry, EU, DMD, Catalysis

Total consideration to Santhera is up to USD 231 million (including equity investment), plus royalty payments from product sales, with near-term cash proceeds for Santhera of USD 126 million.

Key Points: 
  • Total consideration to Santhera is up to USD 231 million (including equity investment), plus royalty payments from product sales, with near-term cash proceeds for Santhera of USD 126 million.
  • Under the terms of the agreement, Santhera will grant Catalyst exclusive commercialization rights to vamorolone in North America, comprising the U.S., Canada and Mexico.
  • At closing, Santhera will receive an upfront cash payment of USD 75 million.
  • Santhera will host a conference call on June 20, 2023, at 14:30 CEST / 13:30 BST / 08:30 EDT to discuss the agreement on vamorolone.