Pricing Reimbursment and Market Acces Newsletter #17

Marketing authorisation for Glybera to expire on 25 October 2017

20 April 2017 — uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that it will not pursue the renewal of the Glybera® (alipogene tiparvovec) marketing authorization in Europe when it is scheduled to expire on October 25, 2017.

Full article here.

Tagrisso receives full approval in the EU

25 April 2017 - AstraZeneca has announced that the European Commission (EC) has granted full marketing authorisation for Tagrisso(osimertinib) 40mg and 80mg once-daily tablets for the treatment of adult patients with locally-advanced or metastatic epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC).

Full article here.

SANOFI AND REGENERON ANNOUNCE FDA APPROVAL OF A NEW ONCE-MONTHLY DOSING OPTION FOR PRALUENT® (ALIROCUMAB).

25 April 2017 — Sanofi and Regeneron Pharmaceuticals have announced that the U.S. Food and Drug Administration (FDA) has approved the companies’ new supplemental Biologics License Application (sBLA) for a once-monthly (every four weeks), 300 mg dose of Praluent® (alirocumab) Injection for the treatment of adults with high low-density lipoprotein (LDL) cholesterol.

Full article here.

Epizyme Announces Tazemetostat Fast Track Designation for Follicular Lymphoma and Plenary Session on Phase 2 NHL Data at ICML

25 April 2017 — Epizyme, a clinical-stage biopharmaceutical company creating novel epigenetic therapies, has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to tazemetostat, the Company’s first-in-class EZH2 inhibitor, for the treatment of patients with relapsed or refractory follicular lymphoma, either wild type EZH2 or with EZH2 activating mutations. Fast Track designation is intended to provide expedited processes for the development and FDA review of drugs that may reduce development time and costs associated with bringing a drug to market.

Full article here.

TESARO Announces Approval of VARUBY® (Oral Rolapitant Tablets) by European Commission

26 April 2017 — TESARO, an oncology-focused biopharmaceutical company, has announced that the European Commission (EC) has approved VARUBY® (oral rolapitant tablets) for the prevention of delayed nausea and vomiting associated with highly and moderately emetogenic cancer chemotherapy in adults. Chemotherapy-induced nausea and vomiting (CINV) is a frequent and debilitating, yet often preventable, side effect of chemotherapy.

Full article here.

Orexigen Therapeutics Announces New Drug Submission for Contrave© in Canada

26 April 2017 — Orexigen Therapeutics, a biopharmaceutical company helping to improve the health and lives of patients struggling with weight loss, has announced that Health Canada has completed its screening phase and accepted for review a New Drug Submission for marketing approval of Contrave® (naltrexone HCl and bupropion HCl). The regulatory submission was filed by Valeant Canada, an affiliate of Valeant Pharmaceuticals International, Inc. If approved, Valeant will market and distribute Contrave in Canada as part of its distributorship agreement with Orexigen, executed in August 2016.

Full article here.

Kiadis Pharma announces filing of marketing authorization with the European Medicines Agency for ATIR101™ in blood cancers

26 April 2017 — Kiadis Pharma, a clinical stage biopharmaceutical company developing innovative products to make bone marrow transplantations for patients suffering from blood cancers and inherited blood disorders safer and more effective, has announced it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead product, ATIR101™. The Company is seeking marketing approval in the European Union for ATIR101™ as an adjunctive treatment in haematopoietic stem cell transplantation (HSCT) for malignant disease.

Full article here.

Clinigen and Onxeo initiate Managed Access programme for belinostat in Europe for patients with peripheral T-cell lymphoma (PTCL)

25 April 2017 — Clinigen Group plc’s Idis Managed Access (‘MA’) division and Onxeo , have agreed to launch a Managed Access programme for belinostat (Beleodaq®) in Europe. Belinostat is for use in patients with relapsed or refractory peripheral T-cell lymphoma (‘PTCL’).

Full article here.

ICE Recommends Amgen’s BLINCYTO® (Blinatumomab) for Adults With Relapsed or Refractory Philadelphia Chromosome-Negative B-Precursor Acute Lymphoblastic Leukaemia

The National Institute for Health and Care Excellence (NICE) has published its Final Appraisal Determination (FAD) recommending BLINCYTO® (blinatumomab) as an option for treating adults with Philadelphia-chromosome-negative (Ph-) relapsed or refractory B-precursor acute lymphoblastic leukaemia (ALL), on the basis of the discount agreed in the patient access scheme. NICE reviewed the data available on the clinical and cost-effectiveness of blinatumomab. Having considered evidence on the nature of the disease and the value placed on the benefits of blinatumomab by people with the condition, those who represent them, and clinical experts, NICE concluded that it is a cost-effective use of NHS resources under the end-of-life criteria.

Full article here.

FDA expands approved use of Stivarga to treat liver cancer

The U.S. Food and Drug Administration today expanded the approved use of Stivarga (regorafinib) to include treatment of patients with hepatocellular carcinoma (HCC or liver cancer) who have been previously treated with the drug sorafenib. This is the first FDA-approved treatment for a liver cancer in almost a decade.

Full article here.

PFIZER’S NEXT-GENERATION ALK/ROS1 INHIBITOR, LORLATINIB, GRANTED BREAKTHROUGH THERAPY DESIGNATION FROM FDA FOR ALK-POSITIVE METASTATIC NON-SMALL CELL LUNG CANCER

27 April 2017 — Pfizer has announced that its investigational next-generation ALK/ROS1 tyrosine kinase inhibitor, lorlatinib, was granted Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with anaplastic lymphoma kinase (ALK)-positive metastatic non-small cell lung cancer (NSCLC), previously treated with one or more ALK inhibitors.

Full article here.

European Commission Approves Bristol-Myers Squibb’s Opdivo (nivolumab) for Squamous Cell Cancer of the Head and Neck in Adults Progressing On or After Platinum-based Therapy

27 April 2017 — Bristol-Myers Squibb Company has announced that the European Commission (EC) has approved Opdivo (nivolumab) as monotherapy for the treatment of squamous cell cancer of the head and neck (SCCHN) in adults progressing on or after platinum-based therapy. Opdivo is the first and only Immuno-Oncology (I-O) treatment that demonstrated in a Phase 3 trial a significant improvement in overall survival (OS) for these patients.

Full article here.