What You Missed From Regulatory Agencies Last Week—July 14-18, 2014

The Medicines Company Receives MAA Renewel from EMA, EC

The Medicines Company announced that the EMA, CHMP, and EC have approved the marketing authorization renewal for Angiox (bivalirudin). Angiox is an anticoagulant for adult patients undergoing percutaneous coronary intervention (PCI). This patient group also includes patients with ST-segment elevation myocardial infarction (STEMI) that are undergoing PCI. The drug is also indicated for the treatment of unstable angina/non-ST segment elevation myocardial infarction.

OPKO Clotting Factor VIIa Earns EU Orphan Drug Designation

The European Committee for Orphan Medicinal Products (COMP) granted OPKO Health three orphan drug designations in Europe for its hemophilia drug. The long-acting version of clotting Factor VIIa for Hemophilia A or B with inhibitors to Factor VIII or Factor IX and congenital Factor VII deficiency received orphan drug designation in the U.S. earlier this year.  The drug has shown the potential to improve quality of life in preclinical studies following intravenous or subcutaneous injection.

Sunovion Pharma Receives Canadian Nod Of Approval

Health Canada has approved Sunovion Pharma’s Aptiom for partial-onset seizures in epileptic patients. Aptiom is a voltage-gated sodium channel inhibitor approved as an adjunctive therapy for partial onset seizures. The approval was based upon three Phase 3 safety and efficacy trials in which patients that had no success with previous treatment encountered significant reductions in seizure frequency vs. placebo. Patients taking Aptiom saw a seizure frequency reduction of 50 percent or more from baseline.

Basilea Mold Infection Treatment Snags EU Orphan Drug Designation

Basilea Pharma antifungal isavuconazole received orphan drug designations in the EU for the treatment of invasive aspergillosis and mucormycosis (zygomycosis). The company plans to submit an MAA for the drug mid-2014. Co-development partner Astellas Pharma recently submitted an NDA to the FDA for isavuconazole in invasive aspergillosis and invasive mucormycosis.

Biliary Tract Cancer Drug Designated Orphan Drug In EU

CellAct Pharma announced that its CAP7.1 first-in-class small molecule compound for the treatment of biliary tract cancers received orphan drug designation. The company is currently enrolling adult patients with refractory biliary tract carcinomas in Germany for a phase 2 proof-of-concept study with CAP7.1. The drug will also be under investigation in a phase 2 study in therapy-refractory non-small and small cell lung cancer.

CTI BioPharma Gains Pixuvri Approval In Israel

The Israel Ministry of Health (MOH) has granted approval to CTI BioPharma’s non-Hodgkin lymphoma drug, Pixuvri. In Israel, the drug is a monotherapy for patients with multiply relapsed or refractory aggressive B-cell non-Hodgkin lymphoma (aggressive B-cell NHL) that have not been subject to more than three rounds of previous treatment.  Neopharm Group will be responsible for marketing and distribution of Pixuvri.  In a separate move, the Dutch Healthcare Authority (NZa) and the healthcare insurance board College voor zorgverzekeringen (CVZ) of the Netherlands approved funding for the drug as an add-on therapy for patients seeking a third or fourth line treatment option.

Pharma Talking Points

According to an EMA press release, the EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended 39 medicines for marketing authorization in the first half of 2014. This is a slight decline from the first half of 2013 when 44 medicines were approved; however it’s an increase when looking at 2012 numbers (33 medicines approved).  Some of these drugs approved include Mekinist (trametinib), Gazyvaro (obinutuzumab), Entyvio (vedolizumab), and Daklinza (daclatasvir). 

A new Fitch Ratings report states that biologics will be playing a larger role in global drug development as NMEs increase. NME approvals in 2014 through the end of May increased above those of the prior 5-month period in 2013. The FDA approved 16 novel drugs for marketing compared to 14 in the first five months of 2013. There were 6 biologics approved during the 2014 period—a sharp increase from 2013 when only 1 was approved.

A Boston Globe article discusses the recent trend of multi-national pharma companies jumping overseas for tax-saving purposes. According to the article, in the past 10 years, 40 pharma companies have moved abroad to save money, while still keeping a majority of their operations in the U.S. However, there are some efforts being undertaken in Congress to keep companies from moving overseas to avoid paying taxes to the U.S. The bill on Capitol Hill proposed a two-year moratorium on tax inversions. The bill would also keep companies from moving their tax address abroad should a significant portion of the business remained in the U.S.

A Wall Street Journal article discusses the role crowdfunding could play in early-stage research for new drugs or diagnostic tests. A new study suggests that crowdfunding could be a “viable approach” used to fund proof-of concept or promising theories. This move, the article says, could also lead to more traditional grants or private investments for funding clinical work down the road. Potential forms of crowd-funding include social media efforts and sponsored participation in sporting events in the name of medical research.