Regulatory approval Archives

News and Trends 20 Apr 2023

FDA approves Genentech’s lymphoma drug

Genentech, a member of the Roche Group, has announced the U.S. Food and Drug Administration (FDA) has approved Polivy (polatuzumab vedotin-piiq) in combination with Rituxan (rituximab), cyclophosphamide, doxorubicin and prednisone (R-CHP). The approval covers the treatment of adult patients who have previously untreated diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS) or high-grade B-cell […]

April 20, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 11 Apr 2023

Sumitomo Pharma Oncology receives orphan drug designation for Ewing sarcoma treatment

Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, said the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for TP-1287, an investigational oral CDK9 inhibitor, for the treatment of Ewing sarcoma. “We are delighted to have received this designation for TP-1287 which underscores the need for additional treatment […]

April 11, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 29 Mar 2023

Pharming immunodeficiency drug given FDA approval

Pharming Group N.V. has announced that the US Food and Drug Administration (FDA) has approved Joenja (leniolisib) for the treatment of activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS) in adult and pediatric patients 12 years of age and older. Pharming’s Joenja, an oral, selective PI3Kδ inhibitor, is the first and only treatment approved in the […]

March 29, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 3 Mar 2023

Orphan drug designation for Laevoroc metabolic immune checkpoint inhibitor

Laevoroc Immunology, a privately-owned, Swiss oncology development company, has announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for immune checkpoint inhibitor LR 09. LR 09 is a novel metabolic immune checkpoint inhibitor, for the treatment of patients with hematological malignancies who are diagnosed with a relapse after allogeneic stem […]

March 3, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 28 Feb 2023

European Commission approves first gene therapy for hemophilia B

The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […]

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

Opinion 28 Feb 2023

Life changing treatments for rare disease – a beacon of hope

By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […]

February 28, 2023 - 6 minutesmins - By External Contributor

News and Trends 24 Feb 2023

Sanofi hemophilia drug gets FDA approval

The U.S. Food and Drug Administration (FDA) has approved Sanofi’s ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl], previously referred to as efanesoctocog alfa, a first-in-class, high-sustained factor VIII replacement therapy. ALTUVIIIO is indicated for routine prophylaxis and on-demand treatment to control bleeding episodes, as well as perioperative management (surgery) for adults and children with hemophilia […]

February 24, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 21 Feb 2023

FDA approves alpha-mannosidosis drug

Chiesi Global Rare Diseases, a business unit of Chiesi Farmaceutici S.p.A., an international research-focused healthcare Group (Chiesi Group), says the U.S. Food and Drug Administration (FDA) has approved Lamzede (velmanase alfa-tycv) for the treatment of non-central nervous system manifestations of alpha-mannosidosis (AM) in adult and pediatric patients. AM is an ultra-rare, progressive lysosomal storage disorder […]

February 21, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

FDA approves GSK drug for recurrent or advanced mismatch repair-deficient endometrial cancer

GSK plc says the US Food and Drug Administration (FDA) has given full approval for Jemperli (dostarlimab-gxly) for the treatment of adult patients with mismatch repair-deficient (dMMR) recurrent or advanced endometrial cancer, as determined by a US FDA-approved test, that has progressed on or following a prior platinum-containing regimen in any setting and are not […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Europe approves childhood epilepsy treatment

UCB’s FINTEPLA (fenfluramine) oral solution has been approved in the European Union (EU) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) as an add-on therapy to other anti-epileptic medicines for patients two years of age and older. The approval by the European Commission (EC) was based on safety and efficacy data from a […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 7 Feb 2023

Endogena Therapeutics retinitis pigmentosa treatment gets FDA fast track designation

The US Food and Drug Administration (FDA) has designated the investigation of Endogena Therapeutics’ EA-2353 for the treatment of retinitis pigmentosa (RP) as a fast track development program. Fast track is a process designed to enable patients to benefit earlier from new drugs for serious conditions. Endogena Therapeutics’ EA-2353 takes a novel, small-molecule approach and […]

February 7, 2023 - 2 minutesmins - By Jim Cornall

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Regulatory approval

FDA approves Genentech’s lymphoma drug

Sumitomo Pharma Oncology receives orphan drug designation for Ewing sarcoma treatment

Pharming immunodeficiency drug given FDA approval

Orphan drug designation for Laevoroc metabolic immune checkpoint inhibitor

European Commission approves first gene therapy for hemophilia B

Life changing treatments for rare disease – a beacon of hope

Sanofi hemophilia drug gets FDA approval

FDA approves alpha-mannosidosis drug

FDA approves GSK drug for recurrent or advanced mismatch repair-deficient endometrial cancer

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Europe approves childhood epilepsy treatment

Endogena Therapeutics retinitis pigmentosa treatment gets FDA fast track designation

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The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

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The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

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