⚡ Top Events Today
Regeneron secures historic FDA approval for first gene therapy for genetic deafness
First-ever FDA approval for a gene therapy treating genetic deafness, which will be made available for free in the U.S.
Intellia reports positive pivotal trial results for CRISPR-based rare disease treatment
The CRISPR-based treatment successfully reduced swelling attacks in patients in a pivotal trial.
FDA places clinical hold on Newron's Phase 3 schizophrenia trial after patient death
The FDA halted U.S. enrollment in the Phase 3 trial following a patient death that occurred outside the United States.
Boehringer's survodutide shows 16.6% weight loss in Phase 3, but trails competitors
The dual GLP-1/glucagon agonist demonstrated a 16.6% weight loss in a Phase 3 trial (NCT06309992).
Astellas halts two Phase 1 candidates and a gene therapy program in pipeline restructuring
The company is discontinuing two Phase 1 drug candidates and a gene therapy program as part of a strategic pipeline 'spring cleaning'.
📁 All Events (14019 stories)
Astellas Pharma has halted two Phase 1 drug candidates and a gene therapy program. This decision is part of a pipeline restructuring, indicating a 'spring cleaning' of its early-stage assets.
Astellas Pharma has halted two Phase 1 drug candidates and a gene therapy program. This decision is part of a pipeline restructuring, indicating a 'spring cleaning' of its early-stage assets.
The FDA has granted historic approval for the first gene therapy for genetic deafness, specifically otoferlin-related hearing loss. This first-of-its-kind genetic medicine will be made available for free in the United States by Regeneron.
The FDA has granted historic approval for the first gene therapy for genetic deafness, specifically otoferlin-related hearing loss. This first-of-its-kind genetic medicine will be made available for free in the United States by Regeneron.
The FDA has placed a clinical hold on Newron Pharmaceuticals' phase 3 schizophrenia trial, halting U.S. enrollment. This decision was made following the death of a patient outside of the United States.
The FDA has placed a clinical hold on Newron Pharmaceuticals' phase 3 schizophrenia trial, halting U.S. enrollment. This decision was made following the death of a patient outside of the United States.
Intellia announced positive results from a pivotal trial of its CRISPR-based treatment for a rare disease. The treatment successfully reduced swelling attacks in patients. The results prompt discussion on the attractiveness of this one-and-done approach for patients and doctors.
Intellia announced positive results from a pivotal trial of its CRISPR-based treatment for a rare disease. The treatment successfully reduced swelling attacks in patients. The results prompt discussion on the attractiveness of this one-and-done approach for patients and doctors.
Boehringer's dual agonist survodutide demonstrated a 16.6% weight loss in a Phase 3 trial. While this result was achieved, it fell short of the competitive bar set by existing drugs. Boehringer aims to differentiate survodutide by targeting glucagon to improve its efficacy in cutting cardiometabolic risk.
Boehringer's dual agonist survodutide demonstrated a 16.6% weight loss in a Phase 3 trial. While this result was achieved, it fell short of the competitive bar set by existing drugs. Boehringer aims to differentiate survodutide by targeting glucagon to improve its efficacy in cutting cardiometabolic
The article lists accelerated approvals for malignant hematology and oncology indications that have been subsequently withdrawn by the FDA. These drugs are no longer FDA-approved.
The article lists accelerated approvals for malignant hematology and oncology indications that have been subsequently withdrawn by the FDA. These drugs are no longer FDA-approved.
AstraZeneca has received FDA approval for Saphnelo for the subcutaneous self-administration in SLE. The approval includes the use of the Saphnelo pen for patients.
AstraZeneca has received FDA approval for Saphnelo for the subcutaneous self-administration in SLE. The approval includes the use of the Saphnelo pen for patients.
The US Food and Drug Administration (FDA) approved a higher-dose version of weekly injectable semaglutide, marketed as Wegovy HD. This approval is for adults with obesity or overweight who have at least one weight-related condition.
The US Food and Drug Administration (FDA) approved a higher-dose version of weekly injectable semaglutide, marketed as Wegovy HD. This approval is for adults with obesity or overweight who have at least one weight-related condition.
Pfizer announced positive Phase 3 data for its drug Elrexfio in patients with relapsed or refractory multiple myeloma. The trial demonstrated a statistically significant and clinically meaningful improvement in progression-free survival.
Pfizer announced positive Phase 3 data for its drug Elrexfio in patients with relapsed or refractory multiple myeloma. The trial demonstrated a statistically significant and clinically meaningful improvement in progression-free survival.
The FDA rejected two new products from AbbVie and Grace Therapeutics due to manufacturing-related concerns. One of the rejected products from AbbVie was described as a prospective Botox offering.
The FDA rejected two new products from AbbVie and Grace Therapeutics due to manufacturing-related concerns. One of the rejected products from AbbVie was described as a prospective Botox offering.
The US Food and Drug Administration (FDA) has approved the first generic versions of dapagliflozin. Dapagliflozin is a sodium-glucose cotransporter 2 (SGLT2) inhibitor, also known by its brand name Farxiga.
The US Food and Drug Administration (FDA) has approved the first generic versions of dapagliflozin. Dapagliflozin is a sodium-glucose cotransporter 2 (SGLT2) inhibitor, also known by its brand name Farxiga.
Sanofi's tolebrutinib has received CHMP backing for approval in Europe for certain multiple sclerosis patients. This development follows a complete response letter from the FDA in December.
Sanofi's tolebrutinib has received CHMP backing for approval in Europe for certain multiple sclerosis patients. This development follows a complete response letter from the FDA in December.
The U.S. Food and Drug Administration (FDA) has approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. This therapy is approved for the treatment of genetic hearing loss and was granted under the National Priority Voucher Program.
The U.S. Food and Drug Administration (FDA) has approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy. This therapy is approved for the treatment of genetic hearing loss and was granted under the National Priority Voucher Program.
Sanofi has received FDA approval for Tzield. This approval allows the drug to be used to delay the onset of stage 3 type 1 diabetes in children as young as one year old who have been diagnosed with stage 2 type 1 diabetes.
Sanofi has received FDA approval for Tzield. This approval allows the drug to be used to delay the onset of stage 3 type 1 diabetes in children as young as one year old who have been diagnosed with stage 2 type 1 diabetes.
Cipla Limited and its subsidiary Cipla USA Inc. announced on April 23, 2026, that they received final U.S. FDA approval for their first AB-rated generic of Ventolin® HFA.
Cipla Limited and its subsidiary Cipla USA Inc. announced on April 23, 2026, that they received final U.S. FDA approval for their first AB-rated generic of Ventolin® HFA.
AbbVie received a Complete Response Letter (CRL) from the U.S. FDA for its Biologics License Application (BLA) for trenibotulinumtoxinE (TrenibotE). The FDA's decision was related to manufacturing processes, and no additional clinical studies were requested.
AbbVie received a Complete Response Letter (CRL) from the U.S. FDA for its Biologics License Application (BLA) for trenibotulinumtoxinE (TrenibotE). The FDA's decision was related to manufacturing processes, and no additional clinical studies were requested.
The FDA has approved MSD's once-daily HIV combination drug, Idvynso. GlobalData analysts forecast a blockbuster future for Idvynso, though they note it is unlikely to displace market dominator Biktarvy.
The FDA has approved MSD's once-daily HIV combination drug, Idvynso. GlobalData analysts forecast a blockbuster future for Idvynso, though they note it is unlikely to displace market dominator Biktarvy.