Ron61 schreef op 12 december 2018 23:17:
[...]
The last two years have been transformational for the Leiden-based specialty company Pharming Group NV. In 2016, it had effectively lost control of the commercialization of its intravenous hereditary angioedema (HAE) product, Ruconest, a recombinant human C1 esterase inhibitor (rhC1INH), produced in rabbits, through a series of licensing deals on both sides of the Atlantic, but now its fortunes have turned.
The drug was approved by the US FDA and in Europe in 2014, but Pharming did not market the drug in either territory, as Valeant Pharmaceuticals International Inc and Swedish Orphan Biovitrum AB (SOBI) held the respective rights. (Pharming had originally partnered Ruconest in North America with Santarus Inc.., which was acquired by Salix Pharmaceuticals Ltd. in 2014. Salix was then itself acquired by Valeant the following year.)
Two years ago, Pharming renegotiated its license with SOBI to regain rights to market Ruconest in 21 countries in Western Europe, North Africa and the Middle East, and paid $60m to reacquire the rights from Valeant to commercialize Ruconest in North America. (Also see "Pharming Regains Rights To Ruconest, Seizing More Control Over Its Destiny" - Scrip, 9 Aug, 2016.)
This series of plays by Pharming are now producing their reward, with its latest financial statement in October showing product revenues up by 74% to €98m, and the Dutch company is now working toward restocking its pipeline. (Also see "Pharming Turns A Profit After Reclaiming Ruconest" - Scrip, 17 May, 2018.)
“The history of Pharming has been mixed,” concedes CEO Sijmen de Vries in an interview with Scrip. The company’s current management team have turned the company into a profit-making enterprise for the first time in its history. “The first 29 years are the worst,” jokes CFO Robin Wright.
Lifecycle Management
The company is now looking forward product-wise and is investigating subcutaneous and intradermal forms of Ruconest, in a concentrated format, for HAE prophylaxis. Ruconest is currently used in the acute setting, and when the FDA showed interest in Phase II data the company had on its use in HAE prophylaxis, it gave the company “a bit of a problem”, recalled Wright, because it was already turning its focus to more competitive routes of administration for both acute treatment and prevention. Using the current formulation twice a week as a preventative measure would not be "ethically defensible" for patients with relatively low levels of attack because of its cost to patients and payers, he said.
PHARMING CEO SIJMEN DE VRIES
This problem was not unique to Ruconest, a report by the Institute of Clinical and Economic Review (ICER), published on Nov. 15, compared the cost effectiveness of prophylaxis HAE treatment of three of Ruconest’s rival therapies; Shire PLC’s newly approved monoclonal antibody Takhzyro (lanadelumab) and its C1 inhibitor Cinryze, alongside CSL Behring’s Haegarda.
The report concluded that while long-term prophylaxis with either of the C1 inhibitors or lanadelumab resulted in fewer acute attacks and improved quality of life for people living with HAE, current pricing of all three treatments exceeds traditional cost-effectiveness thresholds from $50,000 to $150,000 per quality-adjusted life year (QALY) gained. CSL’s Haegarda required the smallest price reduction (28%) to be within traditional thresholds, followed by Takhzyro (34%) and Cinryze (60%).
On the best available treatment today, a patient would need around $550,000 to $600,000 worth of prophylaxis treatment, and a further $100,000 worth of acute treatment for a total of $650,000 to $700,000, Wright explained. By contrast, an average patient treating themselves with the best acute therapy only would cost about $240,000 to $250,000, and they would need far fewer injections. “Ethically speaking its absolutely black and white. Prophylaxis is not a good idea for the system, and not a good idea for the patient unless they are very badly affected, by which we mean four to five attacks a month,” he said.