PDUFA

Human Acellular Vessel™ (HAV™) Biologics License Application Granted Priority Review by U.S. FDA for the Treatment of Vascular Trauma

Retrieved on: 
Friday, February 9, 2024

DURHAM, N.C., Feb. 09, 2024 (GLOBE NEWSWIRE) -- Humacyte, Inc. (Nasdaq: HUMA), a clinical-stage biotechnology platform company developing universally implantable, bioengineered human tissue at commercial scale, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review to Humacyte’s Biologics License Application (BLA) seeking approval of the Human Acellular Vessel (HAV) in urgent arterial repair following extremity vascular trauma when synthetic graft is not indicated, and when autologous vein use is not feasible.

Key Points: 
  • This targeted PDUFA date is based on the Priority Review grant, which is a mechanism reserved by FDA for products that, if approved, would significantly improve the treatment, diagnosis, or prevention of serious conditions.
  • Priority Review applications have a six-month review time instead of ten months for a standard review.
  • The Priority Review aligns with the Regenerative Medicine Advanced Therapy (RMAT) designation granted by the FDA in May 2023 for urgent arterial repair following extremity vascular trauma.
  • The HAV is an investigational product and has not been approved for sale by the FDA or any other regulatory agency.

Madrigal Pharmaceuticals Announces Publication of the Phase 3 MAESTRO-NASH Trial of Resmetirom in the New England Journal of Medicine

Retrieved on: 
Thursday, February 8, 2024

NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.

Key Points: 
  • NASH is a leading cause of liver-related mortality and an increasing burden on healthcare systems globally.
  • Resmetirom received Breakthrough Therapy designation from the FDA and is under review to become the first medicine approved to treat patients with NASH with liver fibrosis.
  • Approximately 50% of patients treated with resmetirom 100 mg with biopsies at Week 52 showed either NASH resolution or fibrosis improvement.
  • More than 80% of patients with biopsies at Week 52 had either fibrosis reversal or no progression of fibrosis.

Ascendis Pharma Reports Fourth Quarter and Full Year 2023 Results

Retrieved on: 
Wednesday, February 7, 2024

Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.

Key Points: 
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • During the fourth quarter of 2024, plan to submit an Investigational New Drug application or similar in adults with achondroplasia.
  • Total revenue for the fourth quarter of 2023 was €137.7 million compared to €22.9 million during the same period for 2022.
  • Ascendis Pharma will also host a conference call and webcast today at 4:30 p.m. Eastern Time (ET) to discuss 2023 financial results.

Bristol Myers Squibb Announces Acceptance of U.S. and EU Regulatory Filings for Neoadjuvant Opdivo (nivolumab) and Chemotherapy Followed by Surgery and Adjuvant Opdivo in Resectable Non-Small Cell Lung Cancer

Retrieved on: 
Wednesday, February 7, 2024

Bristol Myers Squibb (NYSE: BMY) today announced two regulatory acceptances for applications for neoadjuvant Opdivo (nivolumab) with chemotherapy followed by surgery and adjuvant Opdivo for the perioperative treatment of resectable stage IIA to IIIB non-small cell lung cancer (NSCLC).

Key Points: 
  • Bristol Myers Squibb (NYSE: BMY) today announced two regulatory acceptances for applications for neoadjuvant Opdivo (nivolumab) with chemotherapy followed by surgery and adjuvant Opdivo for the perioperative treatment of resectable stage IIA to IIIB non-small cell lung cancer (NSCLC).
  • "Between 30% to 55% of non-small cell lung cancer patients who undergo surgery will experience disease recurrence.
  • The study also showed benefits in key secondary endpoints including pathologic complete response (pCR) and major pathologic response (MPR).
  • Opdivo and Opdivo-based combinations have shown efficacy benefits in the neoadjuvant, adjuvant or perioperative settings across four cancers to date, including lung cancer, bladder cancer, esophageal/gastroesophageal junction cancer and melanoma.

BridgeBio Pharma Announces U.S. Food and Drug Administration (FDA) Acceptance of New Drug Application (NDA) for Acoramidis for the Treatment of Patients with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

Retrieved on: 
Monday, February 5, 2024

PALO ALTO, Calif., Feb. 05, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s New Drug Application (NDA) for acoramidis, an investigational drug for the treatment of ATTR-CM. The application was based on positive results from ATTRibute-CM, the Company’s Phase 3 study designed to evaluate the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR). The FDA has set an action date of November 29, 2024 under the PDUFA. The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.

Key Points: 
  • The FDA has set an action date of November 29, 2024 under the PDUFA.
  • The FDA also notified the Company that it is not currently planning to hold an advisory committee meeting to discuss the application.
  • There is a need for more treatment options that can help fill the significant unmet need that exists for patients today.
  • The Company also received acceptance of its Marketing Authorization Application with the European Medicines Agency and is preparing for additional global regulatory submissions.

ARS Pharmaceuticals to Present Positive Clinical Efficacy Data for neffy® (epinephrine nasal spray) at 2024 AAAAI Annual Meeting

Retrieved on: 
Monday, February 5, 2024

The meeting will be held February 23-26, 2024, in Washington, DC.

Key Points: 
  • The meeting will be held February 23-26, 2024, in Washington, DC.
  • One late-breaking presentation reports efficacy outcomes for the first time with neffy in pediatric patients who developed anaphylaxis symptoms following oral food challenge.
  • The efficacy data demonstrated that 100% of patients responded to a single dose of neffy with a 16-minute median time to complete resolution of anaphylaxis symptoms.
  • Clinical data in this neffy trial is therefore in line with historical clinical response data for injection products.

ImmunityBio Quality-of-Life Study in BCG-Unresponsive Bladder Cancer Trial Indicates Improved Physical Function in the 71% Complete Responders Suggesting a Favorable Risk-Benefit Ratio for N-803 Plus BCG

Retrieved on: 
Monday, February 5, 2024

Overall, participants who achieved a complete response with the novel combination therapy reported better physical function by month six of the study than those who did not achieve a complete response.

Key Points: 
  • Overall, participants who achieved a complete response with the novel combination therapy reported better physical function by month six of the study than those who did not achieve a complete response.
  • “Many current therapies for bladder cancer slow disease progression but can cause debilitating side effects,” said Principal Investigator Karim Chamie, M.D., Associate Professor of Urology at UCLA.
  • Bladder cancer is the 10th most-commonly diagnosed cancer, with approximately 80% of newly diagnosed cases being NMIBC.
  • Safety and efficacy have not been established by any Health Authority or Agency, including the FDA.

Abeona Therapeutics Announces Progress Update on Pz-cel Biologics License Application (BLA)

Retrieved on: 
Thursday, February 1, 2024

CLEVELAND, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced, as part of the review process by the U.S. Food and Drug Administration (FDA) for the Biologics License Application (BLA) for pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB), completion by the FDA of both a Bioresearch Monitoring (BIMO) inspection of Abeona and the BLA mid-cycle review meeting.

Key Points: 
  • The FDA inspector did not issue any observations or FDA Form 483s during the inspection.
  • The formal report from the FDA regarding the BIMO inspection will be received at a later date.
  • Following the BIMO inspection, the BLA mid-cycle review meeting took place on January 25, 2024.
  • The FDA reaffirmed its earlier indication that it does not currently plan to convene an Advisory Committee for pz-cel.

Regulatory Applications Accepted in the U.S. and Japan for Bristol Myers Squibb’s Breyanzi (lisocabtagene maraleucel) in Relapsed or Refractory Follicular Lymphoma (FL) and Relapsed or Refractory Mantle Cell Lymphoma (MCL)

Retrieved on: 
Tuesday, January 30, 2024

In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for Breyanzi to expand into new indications to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) and relapsed or refractory mantle cell lymphoma (MCL) after a Bruton tyrosine kinase inhibitor (BTKi).

Key Points: 
  • In the U.S., the FDA has accepted the company’s two supplemental Biologics License Applications (sBLA) for Breyanzi to expand into new indications to include the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) and relapsed or refractory mantle cell lymphoma (MCL) after a Bruton tyrosine kinase inhibitor (BTKi).
  • The FDA has granted both applications Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of May 23, 2024 for Breyanzi in relapsed or refractory FL and May 31, 2024 for Breyanzi in relapsed or refractory MCL.
  • Japan's MHLW has also accepted Bristol Myers Squibb’s supplemental New Drug Application (sNDA) for Breyanzi for the treatment of relapsed or refractory FL.
  • In both studies, Breyanzi demonstrated a consistent safety profile with no new safety signals reported.

2seventy bio Announces New Strategic Path Forward

Retrieved on: 
Tuesday, January 30, 2024

2seventy bio, in partnership with Bristol Myers Squibb (BMS), is taking actions to return Abecma to commercial growth in 2024.

Key Points: 
  • 2seventy bio, in partnership with Bristol Myers Squibb (BMS), is taking actions to return Abecma to commercial growth in 2024.
  • 2seventy bio and BMS are expanding its Abecma site footprint to enable more patients to access the treatment.
  • Upon closing of the transaction with Regeneron, Chip Baird, chief operating officer, will become chief executive officer of 2seventy bio.
  • After 13 years of service on the bluebird bio and 2seventy bio Board of Directors, Dan Lynch will step down from the Board in June and will continue in an advisory role for 2seventy bio.