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BioMarin's rare disease drug fails Phase 3 trial
The drug failed to meet its primary goal and showed no clinical benefit in a Phase 3 study for the rare genetic disorder ENPP1 deficiency.
AstraZeneca wins FDA approval for hypertension drug baxdrostat
The FDA approved baxdrostat, a first-in-class aldosterone synthase inhibitor (ASI), for the treatment of hypertension.
Alumis halts Phase 3 program for Tepezza rival
The company has halted its drug program, a potential competitor to Amgen's Tepezza, and will not proceed with the planned Phase 3 development.
Enhertu approved for two new indications in early breast cancer
The FDA approved Enhertu for two new indications in HER2-positive early breast cancer, expanding its use to earlier stages of the disease.
Tebentafusp confirms long-term survival benefit in uveal melanoma
The final 5-year analysis of a Phase 3 trial demonstrated a sustained overall survival benefit for tebentafusp in HLA-A*02:01-positive adult patients with metastatic uveal melanoma.
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Tebentafusp demonstrated an overall survival benefit in a phase 3 trial for HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma. It is now the first-line standard of care for this population, with the final 5-year overall survival analysis reported.
Tebentafusp demonstrated an overall survival benefit in a phase 3 trial for HLA-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma. It is now the first-line standard of care for this population, with the final 5-year overall survival analysis reported. | Endpoints: overal
A BioMarin drug, acquired in a buyout, failed to meet its primary goal in a rare disease study for ENPP1 deficiency. This trial outcome has raised doubts about the drug's approval prospects with regulators.
A BioMarin drug, acquired in a buyout, failed to meet its primary goal in a rare disease study for ENPP1 deficiency. This trial outcome has raised doubts about the drug's approval prospects with regulators.
BioMarin Pharmaceutical's enzyme replacement therapy for ENPP1 deficiency showed no clinical benefit in a Phase 3 study. The late-stage trial delivered mixed results for the rare genetic disorder.
BioMarin Pharmaceutical's enzyme replacement therapy for ENPP1 deficiency showed no clinical benefit in a Phase 3 study. The late-stage trial delivered mixed results for the rare genetic disorder.
The FDA's April approvals included a slimmer label for Xpovio, two New Molecular Entities (NMEs), and a gene therapy. An sNDA removed Xpovio's accelerated approval for DLBCL.
The FDA's April approvals included a slimmer label for Xpovio, two New Molecular Entities (NMEs), and a gene therapy. An sNDA removed Xpovio's accelerated approval for DLBCL.
AstraZeneca received FDA approval for its aldosterone synthase inhibitor (ASI) baxdrostat for hypertension. This approval follows an impressive phase 3 showing and contributes to AstraZeneca's goal of reaching $80 billion in revenue by the end of the decade. Additionally, Fasenra also received an approval sweetener.
AstraZeneca received FDA approval for its aldosterone synthase inhibitor (ASI) baxdrostat for hypertension. This approval follows an impressive phase 3 showing and contributes to AstraZeneca's goal of reaching $80 billion in revenue by the end of the decade. Additionally, Fasenra also received an ap
Bio-Thera Solutions' golimumab biosimilars, IMMGOLIS™ and IMMGOLIS INTRI™ (golimumab-sldi), received FDA approval as the first biosimilars to Simponi® and Simponi Aria®. Accord BioPharma will commercialize these products in the United States.
Bio-Thera Solutions' golimumab biosimilars, IMMGOLIS™ and IMMGOLIS INTRI™ (golimumab-sldi), received FDA approval as the first biosimilars to Simponi® and Simponi Aria®. Accord BioPharma will commercialize these products in the United States.
The FDA has approved IMMGOLIS (golimumab-sldi) and IMMGOLIS INTRI (golimumab-sldi) as the first biosimilars to Simponi and Simponi Aria. Accord BioPharma will lead the U.S. commercialization of these drugs. IMMGOLIS is approved for the treatment of adults with moderately to severely active rheumatoid arthritis.
The FDA has approved IMMGOLIS (golimumab-sldi) and IMMGOLIS INTRI (golimumab-sldi) as the first biosimilars to Simponi and Simponi Aria. Accord BioPharma will lead the U.S. commercialization of these drugs. IMMGOLIS is approved for the treatment of adults with moderately to severely active rheumatoi
The FDA has approved Enhertu, developed by Daiichi Sankyo and AstraZeneca, for two new indications in HER2-positive early breast cancer.
The FDA has approved Enhertu, developed by Daiichi Sankyo and AstraZeneca, for two new indications in HER2-positive early breast cancer.
Alumis is exploring strategic alternatives for its drug program, a potential rival to Amgen's Tepezza. The company has decided to halt the program and will not pursue the previously envisaged Phase 3 development.
Alumis is exploring strategic alternatives for its drug program, a potential rival to Amgen's Tepezza. The company has decided to halt the program and will not pursue the previously envisaged Phase 3 development.
Aardvark Therapeutics plans to unblind data from its phase 3 Prader-Willi syndrome program. This decision comes after the FDA imposed a full clinical hold on the company's studies.
Aardvark Therapeutics plans to unblind data from its phase 3 Prader-Willi syndrome program. This decision comes after the FDA imposed a full clinical hold on the company's studies.
The FDA approved Tecentriq's label expansion for post-surgical bladder cancer. This approval was granted alongside Natera's Signatera, which will serve as a companion diagnostic.
The FDA approved Tecentriq's label expansion for post-surgical bladder cancer. This approval was granted alongside Natera's Signatera, which will serve as a companion diagnostic.
On May 15, 2026, the FDA approved atezolizumab (Tecentriq) and atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) by Genentech, Inc. These drugs are approved as adjuvant treatments for adults with muscle invasive bladder cancer (MIBC) after cystectomy who have circulating tumor DNA.
On May 15, 2026, the FDA approved atezolizumab (Tecentriq) and atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza) by Genentech, Inc. These drugs are approved as adjuvant treatments for adults with muscle invasive bladder cancer (MIBC) after cystectomy who have circulating tumor DNA.
The FDA approved Immgolis (golimumab-sldi) and Immgolis Intri (golimumab-sldi) as the first interchangeable biosimilars to Simponi and Simponi Aria (golimumab), respectively. These biosimilars are indicated for the treatment of rheumatoid arthritis and ulcerative colitis.
The FDA approved Immgolis (golimumab-sldi) and Immgolis Intri (golimumab-sldi) as the first interchangeable biosimilars to Simponi and Simponi Aria (golimumab), respectively. These biosimilars are indicated for the treatment of rheumatoid arthritis and ulcerative colitis.
The article highlights ongoing accelerated approvals for drugs and biologics. These approvals target non-malignant hematological, neurological, and other disorder indications and are based on surrogate endpoints.
The article highlights ongoing accelerated approvals for drugs and biologics. These approvals target non-malignant hematological, neurological, and other disorder indications and are based on surrogate endpoints.
Aardvark's rare disease trial has been placed on a full clinical hold by the FDA. The company plans to unblind the trial data following this regulatory action.
Aardvark's rare disease trial has been placed on a full clinical hold by the FDA. The company plans to unblind the trial data following this regulatory action.
Kyowa Kirin has secured FDA approval for a dosing update to the Crysvita prescribing information. This update introduces a new dosing option for adults with X-linked hypophosphatemia (XLH).
Kyowa Kirin has secured FDA approval for a dosing update to the Crysvita prescribing information. This update introduces a new dosing option for adults with X-linked hypophosphatemia (XLH).
Ratura Oncology received a positive recommendation from its Safety Review Committee to continue the Cardioprotection and Anticancer Synergy (CPACS) study. This Phase 3 clinical trial is evaluating a treatment for advanced solid tumor patients.
Ratura Oncology received a positive recommendation from its Safety Review Committee to continue the Cardioprotection and Anticancer Synergy (CPACS) study. This Phase 3 clinical trial is evaluating a treatment for advanced solid tumor patients.
On May 15, 2026, the FDA approved fam-trastuzumab deruxtecan-nxki (Enhertu) for two separate indications in adults with HER2-positive early-stage breast cancer. The first indication is for the neoadjuvant treatment of adults with HER2-positive early-stage breast cancer.
On May 15, 2026, the FDA approved fam-trastuzumab deruxtecan-nxki (Enhertu) for two separate indications in adults with HER2-positive early-stage breast cancer. The first indication is for the neoadjuvant treatment of adults with HER2-positive early-stage breast cancer.
Regenxbio's gene therapy for Duchenne muscular dystrophy successfully met its primary endpoint in a pivotal trial. This positive outcome marks a significant win for the company, which has faced recent challenges including an FDA rejection and clinical hold.
Regenxbio's gene therapy for Duchenne muscular dystrophy successfully met its primary endpoint in a pivotal trial. This positive outcome marks a significant win for the company, which has faced recent challenges including an FDA rejection and clinical hold.
Four cancer drugs received FDA approval in May. These approvals cover both haematological and solid tumour indications.
Four cancer drugs received FDA approval in May. These approvals cover both haematological and solid tumour indications.