EMA, NICE, EC News Roundup: Allergan, Alexion, Innate Pharma, And More

Vifor Pharma’s Velphoro Granted Approved In EU

Vifor Pharma was granted marketing authorization in Europe for Velphoro (sucroferric oxyhydroxide), which is intended for the treatment of Chronic Kidney Disease (CKD) patients that are on hemodialysis or peritoneal dialysis. The drug is a non-calcium, iron-based chewable phosphate binder that demonstrated in a phase 3 trial it could sufficiently control hyperphosphatemia in fewer pills than the current standard of care, sevelamer carbonate. The FDA approved Velphoro for the treatment of patients with CKD that are undergoing dialysis in November 2013, and the drug entered the market in March.

Allergan Ozurdex Receives Approval For Indication Extension

Ozurdex was approved by the European Commission (EC) to treat patients suffering from diabetic macular edema-induced (DME) visual impairment that have not had results from non-corticosteroids and/or have an artificial lens implant. In the EU, the drug is already indicated for the treatment of macular edema in patients with retinal vein occlusion (RVO) and for the posterior segment of the eye characterized as non-infectious uveitis.

EMA Grants Orphan Drug Designations To Cerenis Candidate

CER-001, Cerenis’ candidate for cardiovascular disease, received two orphan drug designations from the EMA for patients with apoA-l deficiency and ABCA1 deficiency in HDL synthesis/maturation pathways. These defects can cause HypoAlphalipoproteinemia (FPHA), which effects the body’s ability to eliminate cholesterol from the blood vessels, in turn causing accelerated atherosclerosis and premature cardiovascular disease. The drug was investigated for safety and efficacy in the SAMBA phase 2 trial enrolling patients with FPHA and it demonstrated its ability to remove cholesterol and reduce vessel wall dimensions of atherosclerotic arteries in those with low HDL cholesterol.

Prosonix MAA Accepted For Review

The U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) is currently assessing the MAA for PSX1001, which is an inhaled generic version of GSK’s Flixotide (EU) and Flovent (U.S.). The Active Pharmaceutical Ingredient (API) is fluticasone propionate, an inhaled corticosteroid, which is indicated to treat asthma. The MHRA’s acceptance for review of the MAA triggered a milestone payment from Mylan, which licensed Prosonix’s inhaled respiratory products PSX1001 and PSX1050, and for which the company has U.S. marketing rights. 

Innate Pharma’s IPH4102 Awarded EU Orphan Drug Designation

The EC designated IPH4102 an orphan drug for the cutaneous T-cell lymphoma (CTCL) indication. A first-in-class cytotoxic anti-KIR3DL2 antibody, IPH4102 is designed to target and eliminate CTCL cancer cells. The candidate will be investigated in a phase 1 trial starting in 2015.

NICE Recommends Availability Of Soliris For aHUS

NICE recommended the commissioning of Alexion’s eculizumab (Soliris) for those with atypical hemolytic uremic syndrome (aHUS). In its draft guidance, NICE recommends that the drug be made available with funding for all patients with aHUS under several key conditions. These conditions include: coordination of eculizumab use through an expert center; monitoring systems that will keep records of those diagnosed with aHUS, those being treated with eculizumab, and the dosage received; a national protocol for starting and stopping treatment with eculizumab; and a research program aimed at establishing when to stop or adjust treatment dosage. The drug is currently only available through an interim policy from NHS England.

Triumeq Wins Marketing Authorization

ViiV Healthcare’s treatment Triumeq received marketing authorization from the EC as a once-daily, single-pill treatment for those suffering from HIV. The drug is a dolutegravir-based regimen combining dolutegravir with avacavir and lamivudine, two nucleoside reverse transcriptase inhibitors (NRTIs). The drug was examined in two SINGLE trials, in which it demonstrated its ability to disrupt the replication of the virus in the body. The drug was also recently approved by the FDA.

Japan’s MHLW Awards Alexion Orphan Drug Designation

Japan’s Ministry of Health, Labor, and Welfare (MHLW) awarded Alexion’s drug candidate asfotase alfa orphan drug designation for patients with hypophosphatasia (HPP). The drug is a first-in-class targeted enzyme replacement therapy which aims to halt the cause of HPP — a disease which can damage bones, cause muscle weakness, seizures, respiratory failure, and even death. The drug is designed to normalize defective metabolic process, in turn preventing or reversing life-long complications from dysregulated mineral metabolism. The drug was recognized by the FDA in 2013 when the regulatory agency deemed it a Breakthrough Therapy. In the EU, the drug’s MAA is currently being assessed under an accelerated assessment program.

IQWiG Gives Nod Of Approval To Janssen’s Olysio

Janssens’s Olysio (simeprevir) received positive news from IQWiG, however, which recommended the drug for the treatment of genotype 1 hepatitis C. This move could serve as a huge boost for the drug, which is already expected to be a huge money maker for the company. Indeed, the drug is expected to bring in close to $2 billion following its first year on the market, PMLive reports.