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Vertex scores EU approval for cystic fibrosis drug Kalydeco in R117H+ children and adolescents

Vertex has revealed that it has secured a new approval from the European Commission for its therapy Kalydeco (ivacaftor) in the treatment of cystic fibrosis (CF) in children and adolescents.

The approval relates specifically to patients at least six months old who weigh at least 5kg whose cystic fibrosis transmembrane conductance regulator (CFTR) gene features the most common residual function mutation, R117H.

Vertex's Kalydeco seizes EU CHMP recommendation for Kalydeco in R117H+ cystic fibrosis

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its recommendation for the approval of Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) in a new indication, Vertex has revealed.

The recommended indication specifically relates to CF patients of at least six months old who weigh at least 5 kg and have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Data revealed on novel CRISPR/Cas9 gene-editing therapy for first two severe haemoglobinopathy patients

Vertex and CRISPR Therapeutics have unveiled the first interim Phase 1/2 data demonstrating the safety and efficacy of the investigational CRISPR/Cas9 gene-editing therapy CTX001. The data concerns the treatment of the first two patients with severe haemoglobinopathies.

The first patient, who has transfusion-dependent beta thalassemia (TDT), received transfusion in the first quarter of this year, generating nine months of follow-up data on the therapy. The individual has the β0/IVS-I-110 genotype and required an annualised rate of 16.5 transfusions per year.

Vertex's cystic fibrosis portfolio to be made available across Northern Ireland and Wales

After drawn out negotiations and much discontent among cystic fibrosis (CF) patients and their advocates, Vertex has announced that a deal has finally been reached that will make its crucial CF medications available to patients across Wales and Northern Ireland.

On 12 November, it emerged that the Regional Pharmaceutical Procurement Service in Northern Ireland reached an agreement with the manufacturer to make available all of its currently licensed CF therapies to patients in the region.

FDA approves Vertex's Trikafta in new indication, now potentially covering 90% of cystic fibrosis patients

The FDA has granted marketing authorisation to Vertex’s Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF), it has emerged.

The approval relates specifically to patients over the age of 12 who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common in the disease.

Vertex cystic fibrosis drugs become available in Scotland after NHS rejection

Two potentially life-changing cystic fibrosis drugs are to become available for a potential pool of 400 patients in Scotland just a month after they were rejected by the NHS.

Boston-based pharmaceutical company Vertex has announced it has signed a five-year deal with the Scottish Government for Orkambi and Symkevi (lumacaftor/ivacaftor) – drugs which help improve lung health, but were to cost about £100,000 per patient.

Scotland turns down Vertex's Orkambi

The Scottish Medicines Consortium (SMC) has turned down Vertex’ cystic fibrosis drug Orkambi on the grounds that it is not good value for money.

Scotland’s cost-effectiveness body rejected the $272,000 a year drug as a treatment for cystic fibrosis in patients aged two and older due to “significant uncertainty around their overall health benefits in the long-term, in relation to their costs.”

Orkambi could benefit 330 people in Scotland, a region which has one of the highest incidences of cystic fibrosis in the UK.

FDA expands Vertex's cystic fibrosis drug Symdeko in paediatric patients

Vertex has announced that the FDA has chosen to expand the existing authorisation on its cystic fibrosis (CF) therapy Symdeko (tezacaftor/ivacaftor).

The drug received marketing approval in February last year in patients over 12 years old who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, or have at least one mutation that is responsive to tezacaftor/ivacaftor; this new approval extends the label to include patients as young as six years old.

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Meanwhile MPs in the House of Commons discussed using Crown Use licensing rules to break Vertex’s IP rights over Orkambi. The discussion marked a new stage in the longstanding stale mate between the US firm and the British government.

MPs discuss breaking Vertex's IP rights to bring Orkambi to UK

MPs yesterday discussed circumventing Vertex’s patent on cystic fibrosis medicine Orkambi (Lumacaftor/ivacaftor) as a means of breaking the deadlock and giving UK patients access to the £104,000 a year drug.

In a meeting at the House of Commons, MPs called on the Government to ramp up the pressure on the US firm, as Parliamentary Under Secretary of State for Health, Seema Kennedy, said she had “a moral obligation” to consider using ‘Crown Use’ licensing laws to break Vertex’s intellectual property (IP).

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