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cystic fibrosis

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

Vertex's Orkambi can be used in even younger cystic fibrosis patients, says European Commission

Vertex has announced that the European Commission has decided to approve a label extension in the region for Orkambi (lumacaftor/ivacaftor) in the treatment of cystic fibrosis (CF), expanding the pool of eligible patients to include children aged between two and five years of age who have two copies of the F508del mutation.

Top Ten most popular articles on Pharmafile.com this week

It's Friday the 13th, but that doesn't change a strong showing from the industry this week as it continued to deliver some remarkable news: the pharma M&A train keeps rolling as the year goes on as Novartis announced it is to shell out $8.7 billion to snatch up gene therapy biotech AveXis, while an exciting partnership is promising to transform the hepatitis C market by offering a drug which boasts a 97% cure rate in trials for as little as $300.

Top Ten most popular articles on Pharmafile.com this week

Some of the biggest news this week concerns companies that are looking to move on parts of their business, but as Pfizer has shown, it is sometimes easier said than done. In addition, two former Kite executives have been unable to resist being drawn back into the field, with a big to make their former technology obsolete. 

Check out the week's ten most popular articles on Pharmafile.com now!

Transforming cystic fibrosis treatment

Published on 05/04/18 at 10:29am

Fred van Goor, Principle Research Fellow at Vertex Pharmaceuticals, discusses his history of work in cystic fibrosis and how the company’s development of Kalydeco succeeded against all odds.

What is your role at Vertex?

I’ve been working at Vertex since 2001 and they’ve had developmental programmes for Cystic Fibrosis (CF) since day one. There have been some big challenges but we’ve been fortunate to have some successes along the way, as well.

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