Regulatory approval Archives

Opinion 13 Jun 2023

How will European Commission proposals on data exclusivity and the bolar exemption affect pharma companies?

By Andrew Sharples, co-head of EIP’s UK litigation team In April, the European Commission published proposed legislation (specifically a new Regulation and a new Directive) aimed at reforming EU legislation in relation to pharmaceuticals. These proposals, if adopted, will affect pharmaceutical regulation in several ways. In relation to IP specifically, they will affect the periods […]

June 13, 2023 - 5 minutesmins - By External Contributor

blood bleeding disorders von Willebrand disease

News and Trends 2 Jun 2023

von Willebrand disease treatment gets FDA orphan drug designation

The U.S. Food and Drug Administration (FDA) has granted Vega Therapeutics, Inc. orphan drug designation for its antibody therapy, VGA039, for the treatment of the rare bleeding disorder, von Willebrand disease (VWD). Vega Therapeutics is a clinical stage biotechnology company developing novel therapies for rare blood disorders. VGA039 is a first-in-class antibody therapy with a […]

June 2, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 26 May 2023

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency (PKD). PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based […]

May 26, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 22 May 2023

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

The U.S. Food and Drug Administration has approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The approval of Vyjuvek was granted to […]

May 22, 2023 - 3 minutesmins - By Jim Cornall

Osteopetrosis ADO2 radiopaedia case ID 8037

News and Trends 16 May 2023

SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis

SiSaf Ltd, an RNA delivery and therapeutics company, has announced that SIS-101-ADO, its siRNA therapeutic for patients with autosomal dominant osteopetrosis Type 2 (ADO2), has been granted orphan drug designation by the U.S. FDA. Also, due to the serious manifestations of this rare skeletal disorder in children, SIS-101-ADO has been granted rare pediatric disease designation […]

May 16, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 15 May 2023

New menopause drug gets FDA approval

The U.S. Food and Drug Administration has approved Astellas Pharma’s Veozah (fezolinetant), an oral medication for the treatment of moderate to severe vasomotor symptoms, or hot flashes, caused by menopause. Astellas Pharma US, Inc.’s Veozah is the first neurokinin 3 (NK3) receptor antagonist approved by the FDA to treat moderate to severe hot flashes from […]

May 15, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 9 May 2023

Fabry disease treatment gets EU marketing authorization

Chiesi Global Rare Diseases and Protalix BioTherapeutics, Inc. have announced that the European Commission (EC) has granted marketing authorization to PRX-102 (pegunigalsidase alfa) in the European Union (EU) for the treatment of adult patients with Fabry disease. “People living with Fabry disease often perceive their disease as burdensome and still experience unmet medical needs,” said […]

May 9, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 4 May 2023

World’s first RSV vaccine for older adults gets FDA approval

GSK plc says the US Food and Drug Administration (FDA) has approved Arexvy (respiratory syncytial virus vaccine, adjuvanted) for the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in individuals 60 years of age and older. It is the first RSV vaccine for older adults to be approved anywhere in […]

May 4, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 2 May 2023

Schizophrenia injection gets FDA approval

Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., and MedinCell have announced the U.S. Food and Drug Administration (FDA) has approved UZEDY (risperidone) extended-release injectable suspension for the treatment of schizophrenia in adults. UZEDY is the first subcutaneous, long-acting formulation of risperidone that utilizes SteadyTeq, a copolymer technology proprietary to MedinCell that controls […]

May 2, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 27 Apr 2023

Biogen ALS drug gets FDA ok

The U.S. Food and Drug Administration (FDA) has approved Biogen’s QALSODY (tofersen) 100 mg/15mL injection for the treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This indication is approved under accelerated approval based on reduction in plasma neurofilament light chain (NfL) observed in patients […]

April 27, 2023 - 5 minutesmins - By Jim Cornall

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

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Regulatory approval

How will European Commission proposals on data exclusivity and the bolar exemption affect pharma companies?

von Willebrand disease treatment gets FDA orphan drug designation

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

SiSaf siRNA therapy gets FDA designations to treat autosomal dominant osteopetrosis

New menopause drug gets FDA approval

Fabry disease treatment gets EU marketing authorization

World’s first RSV vaccine for older adults gets FDA approval

Schizophrenia injection gets FDA approval

Biogen ALS drug gets FDA ok

FDA approves Genentech’s lymphoma drug

Sumitomo Pharma Oncology receives orphan drug designation for Ewing sarcoma treatment

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Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Catalonia: A biotech hub going from strength to strength, with Barcelona at its core

Top biotech deals of February 2025

Grab your free report on 3D organoids and explore their future potential

These 7 biotechs are shaping the next generation of endometriosis care

Connect with decision-makers and researchers worldwide for collaborative opportunities

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Advertise with us

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