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cystic fibrosis

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.

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.

Parents of children with cystic fibrosis start Orkambi buyers club

Parents of children with cystic fibrosis have started an Orkambi buyers’ club to import generic versions of Vertex’s drug from Argentina into the UK.

Frustrated with the stalemate between the NHS and the Massachusetts-based firm, parents of children with cystic fibrosis have come together to import generic versions of Vertex’s £104,000 a year drug into the UK.

The price of access: Can the NHS afford Orkambi?

Published on 29/04/19 at 11:43am

The impasse over access to Vertex’s Orkambi for cystic fibrosis patients in England and Wales has revealed the cracks in the NHS’ regulatory system. Matt Fellows explores where the core issues lie in the debate, and what can be done to prevent future breakdowns in communication for the good of patients.

Vertex destroyed nearly 8,000 packs of Orkambi in 2018

Vertex destroyed nearly 8,000 packs of the $272,000 a year cystic fibrosis drug Orkambi in 2018, the company said in a letter to Sarah Wollaston MP.

The drugmaker, who are locked in negotiations with Britain’s cost effectiveness body NICE, revealed they had destroyed 7,880 packs of Orkambi after the medicines had passed their expiry date.

The revelation came after the US firm was questioned by the Health and Social Care Committee earlier this month.

Vertex doubles Q4 profits, angering cystic fibrosis drug advocates in UK

Despite ongoing controversy surrounding the currently stalled negotiations between Vertex and the NHS to make its cystic fibrosis (CF) drug Orkambi (ivacaftor/lumacaftor) available for patients at a price the health service can afford, the US firm has angered critics in the UK with the announcement of a surge in profits in the last quarter of 2018 – profits it says was driven primarily by “the strong growth in total CF product revenues”.

For the last quarter of 2018, Vertex reported an increase in revenue of 40%, while its profits for the same period more than doubled form $158 mil

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