£11 million-a-year US pharmaceutical boss is accused of trying to ‘blackmail’ Theresa May into buying cystic fibrosis drug

By Stephen Adams and Glen Owen

The boss of a US pharmaceutical giant was last night accused of trying to ‘blackmail’ Theresa May into buying a groundbreaking drug at an eye-watering annual cost of £100,000 per patient.

Thousands of people with the deadly lung condition cystic fibrosis (CF) have been pleading with NHS bosses to strike a deal with Vertex over Orkambi, a new treatment that could extend their lives by years.

The talks collapsed acrimoniously ten days ago, after which Vertex boss Dr. Jeff Leiden wrote to the Prime Minister threatening to scale back the firm’s investment in the UK unless NHS England agreed to buy Orkambi on its terms.

In an extraordinary outburst, Dr. Leiden accused Britain of placing ‘a lower value on the life of a CF patient than other countries’ which have purchased Orkambi, and warned that British jobs relied on his benevolence.
The row comes amid growing anger among health chiefs about how the ‘profiteering’ of drugs companies is heaping pressure on the NHS budget.

The starting price for Orkambi negotiations is thought to be £104,000 per patient per year.

Tory MP Bob Stewart said: ‘Drug companies have every right to turn a profit to fund their vital research and development work, but it is completely immoral to overcharge for a life-saving drug. If the CEO is trying to blackmail the Prime Minister that is utterly disgraceful.’

In his letter to Mrs. May, Dr. Leiden, who received £11 million in salary and stock options last year, wrote: ‘Vertex is heavily committed to the UK with its international HQ in London, an R&D site near Oxford and runs many clinical trials in the NHS. Since 2006, Vertex has invested over £1.2 billion in the UK and now employs 250 people, of whom over 110 are scientists.’

But he added: ‘We are questioning this ecosystem as [the UK] is unable to value life-changing medicines for the patients that need them.’ Without major changes to how new drugs were assessed, he pointedly warned that ‘any future biotech investment in the UK is at significant risk’.

In 2014, Dr. Leiden, who owns a string of waterfront homes in Massachusetts and Florida, faced a shareholder revolt over his ‘exorbitant’ pay package of almost £35 million. Orkambi is the only current therapy that slows the inevitable progression of CF. It could benefit up to half of the 10,500 Britons with the condition.

CF causes lungs to produce too much mucus, reducing their effectiveness. Around half of sufferers die before the age of 47, but Orkambi has been shown to boost lung function by 42 percent.

Catherine Upstone, from Brackley, Northamptonshire, whose daughter Cerys, 14, is one of those who could benefit from Orkambi, said: ‘The two sides need to get back around the table.’

But NHS England boss Simon Stevens recently told MPs that Vertex must first drop its price.

‘Right now, the company is a very long way from acting responsibly,’ he said. ‘The implication of the kinds of prices that are being talked about would either be to deny patients those drugs or to rip off British taxpayers, and neither of those situations is acceptable.’

Vertex’s chief commercial officer Stuart Arbuckle said the firm had made NHS England ‘the best offer in the world, which provides all eligible patients immediate access to our current and future medicines. However, the NHS wants our existing medicines and those coming down the line in the next year for no extra money – this is essentially asking for our medicines for free.’

NHS England said: ‘If Vertex believes they are offering a reasonable deal, they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair.’

Original article here.

‘Sham’ or public interest? ICER suggests 70%-plus discounts on Vertex’s cystic fibrosis drugs

By Angus Liu

Vertex has often talked about its admiration for Gilead, setting the big biotech’s ability to roll out multiple antivirals as a model for its cystic fibrosis endeavor. Now, though, it faces the same pricing issue once pinned on Gilead’s hepatitis C franchise. But the company refuses to play sitting duck.

In a new report, the U.S. cost-effectiveness watchdog the Institute for Clinical and Economic Review argues that Vertex’s cystic fibrosis trio—Kalydeco, Orkambi and newly approved Symdeko—need a huge price cut to achieve cost-effectiveness. Its suggested discount? Over 70%. Continue reading ‘Sham’ or public interest? ICER suggests 70%-plus discounts on Vertex’s cystic fibrosis drugs

New cystic fibrosis drug (Vertex)

https://www.ft.com/content/aa944954-13f4-11e7-b0c1-37e417ee6c76

Combination treatment offers safer option for patients suffering from deadly illness

Vertex Pharmaceuticals said that a combination of two of its medicines had successfully treated cystic fibrosis in a closely watched clinical trial, paving the way for regulators to approve a Continue reading New cystic fibrosis drug (Vertex)

Vertex to get new investment from CFF

Vertex to get new investment from Cystic Fibrosis Foundation

https://www.bostonglobe.com/business/2016/10/14/vertex-get-new-investment-from-cystic-fibrosis-foundation-under-revised-deal/XklbRNebBa1nOhgt4WOqFI/story.html

Vertex Pharmaceuticals Inc. will receive an upfront payment of $75 million and additional payments of up to $6 million a year for the development of new cystic Continue reading Vertex to get new investment from CFF

Modern Love Column in NY Times–Worth Reading

My husband and I usually read the Social Qs column on Sundays in the New York Times. Then we glance at the rest of the Styles Section. He saw that the Modern Love column was about a double lung transplant that almost destroyed a Continue reading Modern Love Column in NY Times–Worth Reading

Nivalis Therapeutics Announces First Patient Dosed in Second Phase 2 Study of N91115 for Treatment of Cystic Fibrosis

https://globenewswire.com/news-release/2016/05/16/840427/0/en/Nivalis-Therapeutics-Announces-First-Patient-Dosed-in-Second-Phase-2-Study-of-N91115-for-Treatment-of-Cystic-Fibrosis.html

Nivalis Therapeutics, Inc. (NASDAQ:NVLS), a clinical stage pharmaceutical company focused on treating people with cystic fibrosis (“CF”), today announced the first patient was dosed in a second Phase 2 clinical study of Continue reading Nivalis Therapeutics Announces First Patient Dosed in Second Phase 2 Study of N91115 for Treatment of Cystic Fibrosis

The Paradox of Precision Medicine

http://www.scientificamerican.com/article/the-paradox-of-precision-medicine/

Precision medicine sounds like an inarguably good thing. It begins with the observation that individuals vary in their genetic makeup and that their diseases and responses to medications differ as a result. It then aims to find the right drug, for the right patient, at the Continue reading The Paradox of Precision Medicine

Nivalis’ N91115 Gets Orphan Drug Status for Cystic Fibrosis

Nivalis Therapeutics, Inc. announced that the FDA has granted orphan drug designation to its lead candidate, N91115, for the treatment of patients suffering from cystic fibrosis. The stock surged 15.5% on the news. Continue reading Nivalis’ N91115 Gets Orphan Drug Status for Cystic Fibrosis

Galapagos advances CF clinical development

  • Initiation of Phase 1 study with corrector GLPG2222, earning Galapagos a $10 million milestone payment from AbbVie
  • Expansion of CF portfolio with three novel candidate drugs, with aim to create the most effective combo

Mechelen, Belgium; 19 January 2016: Galapagos NV (Euronext & NASDAQ: GLPG) provides an update on progress made in the cystic fibrosis (CF) programs. Galapagos and AbbVie (NYSE: ABBV) aim to develop a triple CFTR combination therapy to address 90% of patients with CF. In order to bring a more effective therapy to patients, the companies plan to develop multiple candidates and backups for each of the three components (1st generation correctors, next generation correctors, and potentiators) with the goal of identifying a potential triple combination. In addition to our potentiator and corrector that are being tested in the clinic, a total of four additional compounds have the potential to reach clinical development in the CF program this year. Continue reading Galapagos advances CF clinical development

Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=949601

-Approximately 25,000 people with cystic fibrosis worldwide currently eligible for treatment with ORKAMBI® (lumacaftor/ivacaftor) or KALYDECO® (ivacaftor); Vertex Continue reading Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases