Priority review

Sarepta Therapeutics Submits Efficacy Supplement to Expand the ELEVIDYS Label to include Duchenne Muscular Dystrophy Patients without Restriction to Age or Ambulatory Status

Retrieved on: 
Friday, December 22, 2023

The supplement was submitted to the U.S. Food and Drug Administration (FDA) with a request for Priority Review.

Key Points: 
  • The supplement was submitted to the U.S. Food and Drug Administration (FDA) with a request for Priority Review.
  • An efficacy supplement is a submitted request for a proposed change to an approved product’s labeling, including adding or modifying an indication previously filed with the FDA.
  • Sarepta has also completed the EMBARK postmarketing requirement (PMR) and submitted the PMR to FDA requesting conversion from accelerated approval to traditional approval.
  • Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world.

Patritumab Deruxtecan Granted Priority Review in the U.S. for Certain Patients with Previously Treated Locally Advanced or Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

Retrieved on: 
Friday, December 22, 2023

The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is June 26, 2024.

Key Points: 
  • The Prescription Drug User Fee Act (PDUFA) date, the FDA action date for their regulatory decision, is June 26, 2024.
  • The Priority Review follows receipt of Breakthrough Therapy Designation granted by the FDA in December 2021.
  • RTOR allows the FDA to review components of an application before submission of the complete application.
  • Twelve patients (5.3%) had confirmed treatment-related interstitial lung disease (ILD) as determined by an independent adjudication committee.

FDA Grants Priority Review to Xolair (omalizumab) for Children and Adults With Food Allergies Based on Positive National Institutes of Health Phase III Study Results

Retrieved on: 
Tuesday, December 19, 2023

If approved, people taking Xolair would still need to avoid foods they are allergic to (commonly referred to as “food avoidance”).

Key Points: 
  • If approved, people taking Xolair would still need to avoid foods they are allergic to (commonly referred to as “food avoidance”).
  • The filing acceptance is based on positive interim analysis results from Stage 1 of the National Institutes of Health (NIH)-sponsored pivotal Phase III OUtMATCH study evaluating Xolair in patients allergic to peanuts and at least two other common foods.
  • If approved, Xolair would be the first medicine to reduce allergic reactions to multiple foods following an accidental exposure.
  • “We are proud to partner with the National Institutes of Health and leading research institutions on this groundbreaking study.

Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments

Retrieved on: 
Monday, November 13, 2023

Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024

Key Points: 
  • Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024
    BOSTON and LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced several business accomplishments along with its financial results for the quarter ended September 30, 2023.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.
  • The company had approximately 227.3 million ordinary shares, equivalent to 22.7 million American Depositary Shares, outstanding as of September 30, 2023.
  • As a result of the anticipated acquisition by Kyowa Kirin, Orchard Therapeutics has removed its financial guidance around Libmeldy revenue and anticipated cash runway.

Abeona Therapeutics Reports Third Quarter 2023 Financial Results and Corporate Developments

Retrieved on: 
Monday, November 13, 2023

CLEVELAND, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (Nasdaq: ABEO) today reported financial results for the third quarter of 2023 and provided corporate updates.

Key Points: 
  • The FDA also agreed that retroviral vector manufactured at Abeona and Indiana University appear comparable based on the data that Abeona provided in its briefing book.
  • If accepted with Priority Review, Abeona expects potential BLA approval in the second quarter of 2024.
  • Abeona estimates that its cash and cash equivalents, restricted cash and short-term investments as of September 30, 2023 are sufficient resources to fund operations into the fourth quarter of 2024.
  • Abeona Therapeutics will host a conference call and webcast today, November 13, 2023, at 8:30 a.m.

Madrigal Pharmaceuticals Presents New Data from the Phase 3 MAESTRO-NASH Trial Demonstrating Broad Treatment Effects of Resmetirom on Noninvasive Measures of Liver Health

Retrieved on: 
Friday, November 10, 2023

Resmetirom is a liver-directed thyroid hormone receptor (THR)-β agonist oral therapy that is designed to target key underlying causes of NASH.

Key Points: 
  • Resmetirom is a liver-directed thyroid hormone receptor (THR)-β agonist oral therapy that is designed to target key underlying causes of NASH.
  • It is the only investigational therapy for NASH that has achieved both fibrosis improvement and NASH resolution primary endpoints in a Phase 3 trial.
  • Resmetirom also reduced levels of LDL cholesterol and other lipids that are associated with heart disease.
  • Resmetirom is an investigational therapy and has not been approved by the FDA or any other regulatory authority.

Valneva Announces U.S. FDA Approval of World’s First Chikungunya Vaccine, IXCHIQ®

Retrieved on: 
Friday, November 10, 2023

Continued approval for this indication is contingent upon verification of clinical benefit in confirmatory studies.

Key Points: 
  • Continued approval for this indication is contingent upon verification of clinical benefit in confirmatory studies.
  • As sponsor of the first chikungunya vaccine approved in the U.S., Valneva has received a Priority Review Voucher (PRV) from the FDA, which it intends to monetize to help finance its research and development (R&D) programs.
  • With this U.S. approval, IXCHIQ® becomes the world’s first licensed chikungunya vaccine available to address this unmet medical need and the third vaccine Valneva1 has brought from early R&D to approval.
  • Initial safety data from this trial were included in the submission to the European Medicines Agency (EMA) in October 202313.

Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the Treatment of Niemann-Pick Disease Type C1

Retrieved on: 
Monday, December 18, 2023

N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, added, “We view our Type C meeting with FDA as a catalytic and transformational milestone for our ongoing pivotal Phase 3 NPC program.

Key Points: 
  • N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, added, “We view our Type C meeting with FDA as a catalytic and transformational milestone for our ongoing pivotal Phase 3 NPC program.
  • For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960 .
  • Cyclo Therapeutics received Orphan Drug Designation for Trappsol® Cyclo™ to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S.
  • The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the U.S. upon marketing authorization.

AnHeart Therapeutics and Innovent Announce China’s NMPA Has Granted Taletrectinib (ROS1 Inhibitor) Priority Review Designation

Retrieved on: 
Monday, December 18, 2023

The CDE accepted the NDA in November 2023.

Key Points: 
  • The CDE accepted the NDA in November 2023.
  • “Taletrectinib’s Priority Review Designation reinforces the urgent need for patients with ROS1-positive NSCLC who were previously treated with ROS1 TKIs in China and the significant clinical advance that taletrectinib may represent for these patients,” said Bing Yan, MD, Chief Medical Officer, AnHeart.
  • “We look forward to working with the regulatory authorities as they conduct their review of taletrectinib to hopefully bring this breakthrough investigational therapy to patients as soon as possible.”
    “Taletrectinib’s potential as a best-in-class precision therapy is further underscored by the Priority Review Designation,” said Dr. Hui Zhou, Senior Vice President of Innovent.
  • Priority Review Designation allows the CDE to prioritize the review process and evaluation resources for NDAs, which helps accelerate the market access of innovative drugs.

bluebird bio Announces FDA Approval of LYFGENIA™ (lovotibeglogene autotemcel) for Patients Ages 12 and Older with Sickle Cell Disease and a History of Vaso-Occlusive Events

Retrieved on: 
Friday, December 8, 2023

LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.

Key Points: 
  • LYFGENIA is a one-time gene therapy that has the potential to resolve vaso-occlusive events and is custom-designed to treat the underlying cause of sickle cell disease.
  • “LYFGENIA has the potential to have a transformational impact for patients who currently live under the shadow of unpredictable and debilitating vaso-occlusive events.
  • The burden associated with VOEs is pervasive and can affect every aspect of life for patients and their families and caregivers.
  • A replay of the webcast will be available on the bluebird website for 90 days following the event.