FDA approves first gene therapy for Haemophilia B
Hemgenix, the first gene therapy for adults with Haemophilia B has been approved by the US Food and Drug Administration (FDA).
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Hemgenix, the first gene therapy for adults with Haemophilia B has been approved by the US Food and Drug Administration (FDA).
The FDA gives clinical trial clearance to 3D-printed colon-targeted oral drug for ulcerative colitis, to help improve safety of the dosage release.
The FDA has accepted priority review of epcoritamab for adults with relapsed/refractory large B-cell lymphoma, which could be the first subcutaneous bispecific antibody for large B-cell lymphoma.
The European Commission has approved Enjaymo®, the first-and-only approved therapeutic for haemolytic anaemia in adults with cold agglutinin disease.
The US Food & Drug Administration (FDA) has approved the first-in-human gene therapy trial for heart failure patients with preserved ejection fraction.
The US Food and Drug Administration (FDA) has approved Tzield, the first drug that can help delay the onset of stage 3 type 1 diabetes in patients over eight years old with stage 2 type 1 diabetes.
Upstaza™, approved for UK patients 18 months and over, is the first gene replacement therapy infused directly into the brain for the treatment of AADC deficiency.
New therapy Mobocertinib, recommended by NICE, helps slow growth of EGFR-positive non-small-cell lung cancer in platinum-based chemo-treated patients.
Monkeypox oligos - Primers & Probes for Research & Commercial use.
The International Society of Pharmaceutical Engineering’s GAMP® 5 guidelines steer validation practices for pharma companies to meet computerized system expectations. Members of the GAMP steering committees, Lorrie Schuessler, Charlie Wakeham and Stephen Ferrell, share the some of the key changes in the second edition update and how these should be…
Under the revised 2022 WHO Biosimilars Guideline, IGBA says regulatory authorities can confidently revaluate biosimilar access requirements.
The Committee for Medicinal Products for Human Use (CHMP)’s November meeting consisted of approval recommendations for four medicines, including a biosimilar for osteoporosis and a new COVID-19 vaccine.
The EC has authorised the first UL97 protein kinase inhibitor for adults with post-transplant refractory cytomegalovirus infection.
Minoryx Therapeutics and the Neuraxpharm Group enter into a license agreement for the European rights to leriglitazone, which is currently under EMA review for the orphan indication X-linked Adrenoleukodystrophy (X-ALD).
Keith A Zullow and Jenny J Zhang from Goodwin Procter examine upcoming US pharmaceutical regulation and how, if passed, it could impact European pharma/biopharma looking to access the US market.