European drug regulator comes out of hibernation in new Dutch headquarters, rivaling the FDA with rulings; well, just for one week.

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It may have taken nearly 6 months and perhaps a Fourth of July holiday-shortened work week here in the US, but the European Medicines Agency (EMA) finally issued some new rulings recently, almost as many as the Food and Drug Administration (FDA).

Back in late January, the EMA, one of the biggest EU regulators and one of the first casualties of Brexit, closed its doors in the UK for the last time with the loss of 900 jobs. The EMA evaluates medicines throughout the EU but was forced to relocate to the Dutch capital because pharmaceuticals regulation should be conducted in a member state. Amsterdam won the bid to host the agency in 2017, The Guardian notes.

The EMS’s departure from London was mourned widely as it marks not just the loss of highly skilled jobs but the UK’s central place in pharmaceutical evaluation and monitoring. At the time, EMA executive director, Guido Rasi, expressed his thanks to the UK, “for its contribution to the work of the Agency and for having been a gracious host of EMA since 1995.”

The decision made by the EU to move the EMA was a body blow to those who had spent a lifetime charting the progress of medicines through evaluation and testing cycles.

Here’s the roundup of last week’s regulatory action, beginning with the US drugs and medical devices agency:

  • The FDA has granted accelerated approval to the oral therapy selinexor (Xpovio), from Karyopharm Therapeutics Inc. (Newton MA), in combination with the corticosteroid dexamethasone, for the treatment of adults with relapsed/refractory multiple myeloma (RRMM) who are out of treatment options. Karyopharm immediately set a list-price for its blood-cancer treatment at $22,000 per month.


  • Elsewhere, the FDA has approved an enteric-coated delayed-release formulation of tiopronin (Thiola EC), from Retrophin Inc. (San Diego), for treatment of cystinuria, a rare inherited disorder that causes an increase in cystine levels in the urine, leading to recurring cystine kidney stones. Thiola EC tablets can be taken with or without food, “an advancement over the original formulation (Thiola) which has limiting food restrictions,” said Eric Dube, PhD, CEO of Retrophin.


  • The FDA approved a 20% immune globulin solution for subcutaneous injection (Xembify), from Grifols SA (Barcelona), to treat primary immunodeficiencies in patients as young as 2 years old, according to a company statement. The approval includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency, X-linked agammaglobulinemia, Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. The company plans to launch Xembify in the US in the last quarter of 2019.


  • And the FDA said three deaths had been reported related to Edwards Lifesciences Corp.’s (Irvine CA) recall of some of its heart devices. The agency said the company’s voluntary recall of its IntraClude intra-aortic occlusion device in May has now been classified by the FDA as Class I, the strictest recall, where use of faulty devices may cause serious injury or death. The FDA said that recall affects more than 750 devices in the US. The recall was due to a risk of balloon rupture during use of the devices, the FDA said, adding the company received 22 complaints related to the device. The IntraClude is used in patients undergoing cardiopulmonary bypass, where the device temporarily takes over the functions of the heart and lungs during surgery.

Across the Pond:

  • Alexion Pharmaceuticals Inc. (New Haven CT) said that the European Commission approved Ultomiris (ravulizumab) for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Specifically, the long-acting C5 complement inhibitor is authorized for adults with PNH with hemolysis with clinical symptoms indicative of high disease activity and also for adults who are clinically stable after having been treated with Alexion’s Soliris (eculizumab) for at least the past six months.


  • The European Commission has approved the fixed-dose combination of dolutegravir and lamivudine (Dovato), from London-based ViiV Healthcare, for HIV-1 infection. Dovato contains 50 mg dolutegravir, an integrase inhibitor, and 300 mg lamivudine, a nucleoside analogue. It is indicated for treatment-naïve adults and children older than 12 years weighing at least 40 kg with no known or suspected resistance to the integrase inhibitor class or lamivudine. The European Medicines Agency’s Committee for Medicinal Products for Human Use recommended approval in April.


  • The EMA’s Committee for Medicinal Products for Human Use (CHMP) has recommended angiotensin II acetate injection (Giapreza), from La Jolla Pharmaceutical Co. (San Diego), for refractory hypotension in adults with septic or other distributive shock who remain hypotensive despite adequate volume restitution and application of catecholamines and other available vasopressor therapies. Giapreza mimics the body’s endogenous angiotensin II regulatory peptide that is central to the renin–angiotensin–aldosterone system to increase blood pressure.


  • The UK’s National Institute for Health and Care Excellence (NICE) published final guidance recommending NHS reimbursement of Pfizer Inc.’s (NYC) Vizimpro (dacomitinib) as a first-line treatment option for adults with locally advanced or metastatic non-small-cell lung cancer (NSCLC), who have tested positive for the EGFR mutation. The regulator’s previous decision deemed that the once-daily tyrosine kinase inhibitor, which was approved in Europe in April, was not a cost-effective use of NHS resources.


  • NICE also published guidance on Friday recommending against use of AstraZeneca PLC’s (London) Tagrisso (osimertinib) for the same patient population–namely, NSCLC, who have tested positive for the EGFR mutation–because the cost-effectiveness estimates were above what it normally considers an acceptable use of NHS resources. AstraZeneca said it will appeal the decision, with CEO Pascal Soriot saying, “it is time for a comprehensive review of how NICE values innovative medicines.”


  • And Sanofi SA (Paris) and Regeneron Pharmaceuticals Inc.’s (Tarrytown NY) Libtayo was two for two last week with European officials. One day after the European Commission cleared its PD-1 drug to treat cutaneous squamous cell carcinoma, England’s cost watchdog signed off on an access agreement for the product in adults whose disease can’t be treated with surgery or radiotherapy. But it’s not an outright win for Libtayo at the National Institute for Health and Care Excellence (NICE). The cost-effectiveness gatekeeper will add the product to its Cancer Drugs Fund rather than cover it for routine use on the National Health Service. And as usual, NICE has some conditions. Patients should receive Libtayo until their disease progresses or they reach the two-year mark- whichever comes sooner.
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