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- PMLiVE

Beta thalassaemia

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FDA grants fast track designation to Nurix’s BTK degrader for non-Hodgkin lymphomas

CLL is one of the most common types of leukaemia in adults, with around 18,740 new cases in the US last year

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Takeda’s HyQvia granted FDA approval to treat rare neuromuscular disorder CIDP

Approximately five to seven people per 100,000 in the US are affected by the disorder

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Sanofi/Regeneron’s Dupixent gets US label update for atopic dermatitis

Dupixent’s label now includes data supporting its use in hand and foot atopic dermatitis

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FDA approves Vertex/CRISPR’s Casgevy gene therapy for beta thalassaemia

The companies estimate that approximately 1,000 patients in the US will be eligible for the one-time treatment

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Merck granted FDA approval for Keytruda combination in cervical cancer

The decision marks the third FDA-approved indication for Keytruda in cervical cancer

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FDA authorises Florida’s request to import cheaper prescription drugs from Canada

The first-of-its-kind importation programme is expected to save the state up to $180m in its first year

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AstraZeneca and Ionis’ Wainua granted FDA approval for rare disease ATTRv-PN

ATTRv-PN leads to peripheral nerve damage with motor disability within five years of diagnosis

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FDA establishes advisory committee for genetic metabolic disease treatments

The committee will evaluate the potential benefits and risks of new therapies for diagnosis, prevention and treatment

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CHMP recommends gene therapy for beta thalassemia and sickle cell disease

If approved, Casgevy will be the only genetic therapy in the EU to treat these conditions

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FDA grants fast track designation to Candel’s immunotherapy in pancreatic cancer

The disease is the fourth leading cause of cancer-related death in the US, accounting for 3.3% of new cancer cases

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FDA approves two cell-based gene therapies for sickle cell disease

Vertex/CRISPR and bluebird bio’s gene therapies have been approved to treat patients aged 12 years and older

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Novartis’ Fabhalta approved by FDA as first oral monotherapy for rare blood disease PNH

The rare blood disease is estimated to affect only ten to 20 people per million worldwide

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