What You Missed From The Regulatory Agencies Last Week—August 4-9, 2014

PTC Therapeutics DMD Drug Earns Conditional EU Approval

The European Commission (EC) has granted conditional marketing approval for PTC Therapeutics’ Translarna in non-mutation Duchenne Muscular Dystrophy (nmDMD). The drug is indicated for ambulatory patients 5 years old and up. The drug will be marketed in 28 countries in the EU, as well as in Iceland, Liechtenstein, and Norway. The company is required to complete its phase 3 nmDMD trial (ACT DMD) and submit safety and efficacy results. The EC took into account the performance of the drug in 174 patients in a 48-week study examining the drug’s effect on ambulation.

EMA Grants AbbVie’s ABT-414 Orphan Drug Designation

AbbVie’s investigational compound ABT-414 has received Orphan Drug Designation in the treatment of glioblastoma multiforme. The compound, an anti-epidermal growth factor receptor antibody drug conjugate is carried through the blood stream and releases its cytotoxic agent once it’s inside the targeted cancer cell. Apart from being in a phase 1 clinical program for patients with recurrent or unresectable glioblastoma multiforme, the drug is also being investigated as a treatment for squamous cell tumors.

Raptor Drug Gains EU Orphan Drug Designation

Cysteamine Bitartrate (API RP103) was designated an orphan drug in the EU for Huntington’s Disease. The drug previously received a positive recommendation from the EMA’s Committee for Orphan Medicinal Products (COMP) in June. This spring, Raptor Pharma announced that RP103 has been holding its own through the ongoing CYST-HD clinical trial for patients with early-stage Huntington’s Disease. The EC took into account the results from the 18-month CYST-HD study as support for its orphan drug application.

EMA Grants Cancer Drug Avastin Approval In EU

Ovarian cancer drug Avastin earned approval from the EC last week. The drug, in combination with paclitaxel, topotecan, or pegylated liposomal doxorubicin chemotherapy, is indicated for women with recurrent, platinum-resistant ovarian cancer. The EC based its approval off of the Phase 3 AURELIA study that examined the effects of Avastin when paired with chemotherapy vs. chemotherapy alone. The drug, put forth by Roche, is currently approved and used in Europe for the treatment of breast, colorectal, and lung cancer. In 2011, the drug was approved as a treatment for women with advanced ovarian cancer.

Pfizer’s Enbrel Approved In EU For Additional Indication

Following the most recent decision by European regulators, tumor necrosis factor inhibitor Enbrel is now indicated for the treatment of severe non-radiographic axial spondyloarthritis (nr-axSpA)— a subtype of the inflammatory disease axial spondyloarthritis (axSpA).  The drug is intended for those in the early stages of the disease. Clinical data showed that the drug demonstrated improvements in measures of disease activity and function and decreased inflammation after 12 weeks.

Health Canada Authorizes Celgene’s Abraxane

Following review of the results of a phase 3 MPACT (Metastatic Pancreatic Adenocarcinoma Clinical Trial), Health Canada has approved Abraxane for injectable suspension. The treatment is indicated for adults with metastatic pancreatic cancer and is a victory for Canadian patients, considering this is the first approved treatment for this disease in nearly 20 years. The MPACT study investigated the drug’s impact on overall survival in 861 chemotherapy-naïve patients, demonstrating the efficacy of Abraxane plus gemcitabine in improving median overall survival.

EMA Accepts Pfizer’s Application For Prevenar 13

Pfizer is looking to extend the indication of Prevenar 13 to include the prevention of pneumonia caused by the 13 pneumococcal serotypes contained in the vaccine. The application was supported by positive results of the Community-Acquired Pneumonia Immunization Trial in Adults (CAPiTA), which showed that the vaccine significantly reduced vaccine-type pneumococcal  community-acquired pneumonia (CAP). The drug is currently approved in Europe to prevent pneumococcal disease. The company also submitted an sBLA to the FDA in order to add data about the drug’s efficacy in treating older adults to the prescribing info.

Hemophilia RNAi Therapeutic Designated Orphan Drug In EU

Alnylam Pharmaceuticals announced that the EMA granted ALN-AT3 orphan drug designation for the treatment of hemophilia A and B. The therapeutic is currently in development to be a subcutaneously administers RNAi therapeutic aimed at targeting antithrombin (AT) in hemophilia and other bleeding disorders. ALN-AT3 is currently being investigated in a multinational phase 1 trial, and the company has presented data showing that the treatment significantly lowered AT and boosted thrombin generation.

Atox Bio NSTI Treatment Named EU Orphan Drug

AB103 (Sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH) was granted orphan drug designation in the EU for necrotizing soft tissue infections (NSTI). There are currently no approved treatments for NSTI, which means patients are often subject to repeated surgical debridement, resuscitation, and antibiotics. AB103 is a novel immunomodulator that binds to the CD28 dimer interface and controls inflammation. It modulates the host’s immune system; it does not inhibit it during course of treatment. The drug showed efficacy in a phase 2 study.

EC Approves Hemophilia A Drug Nuwiq

Octapharma announced that the European Commission (EC) has approved Nuwiq for the treatment and prevention of bleeding in all age groups suffering from hemophilia A. Nuwiq is produced in a human cell line and does not contain any human or animal additives. Nuwiq, or human cell line recombinant FVIII, aims to eliminate the need for frequent infusions to prevent bleeding episodes. FVIII was examined in three pivotal trials and demonstrated positive results in all three of these trials.