News Feature | August 28, 2014

What You Missed From Regulatory Agencies Recently – August 18-28, 2014

By Anna Rose Welch, Editor, Biosimilar Development
Follow Me On Twitter @AnnaRoseWelch

NICE Gives Nod Of Approval To Celgene’s Revlimid

NICE has published final draft guidance stating that Celgene’s Revlimid for myelodysplastic syndromes will be available on the NHS in England. The recommendation applied to people with suffering from a specific type of myelodysplastic syndrome that is characterized by an isolated deletion 5q cytogenetic abnormality. The drug is previous available for those with multiple myeloma. NICE previously published negative guidance for the drug in the treatment of this illness in 2013 on the basis of the drug’s high price — 3,780 pounds per month. However, the drug will be available on the NHS for this indication through a patient access scheme. The company has also provided the additional necessary information to make the drug available to the NHS for a lower cost.

Swiss Drug Authority Approves Venus Remedies Oncology Drug

Venus Remedies earned a marketing authorization from the Swedish drug regulatory agency, Swissmedic, for its oncology drug Gemcitabine. The approval ensures the drug’s launch next quarter in Switzerland. The company has received more than 29 marketing approvals for its Gemcitabine injection in a number of different countries. The drug, which treats a number of cancers including lung, pancreas, breast, and bladder cancer, is currently available in the U.K., Poland, and Germany.

EMA To Review Basilea’s MAA

Basilea Pharma announced that the EMA had accepted its Marketing Authorization Application (MAA) for isavuconazole for review. Should the application be accepted, the drug would be indicated for the treatment of invasive aspergillosis and mucormycosis (zygomycosis) in adults and would be available in the 28 member states of the EU, as well as in Iceland, Liechtenstein, and Norway. Isavuconazole is an investigational, once-daily intravenous and oral broad-spectrum antifungal catered towards those who are afflicted by fungal infections because of a compromised immune system (i.e. cancer patients undergoing chemotherapy).  

Cubist MAA Accepted For Review By EMA

Cubist’s investigational antibiotic, ceftolozane/tazobactam, was accepted for review by the EMA. Cubist aims to have the antibiotic approved for the treatment of complicated urinary tract infections and complicated intra-abdominal infections. A decision, which will take into account positive data gleaned from two pivotal Phase 3 trials, is expected to be made during the second half of 2015. In the Phase 3 trials, the drug showed efficacy fighting Gram-negative bacteria, including Pseudomonas aeruginosa and extended-spectrum beta-lactamase (ESBL)-producing Escherichia coli (E. coli) and Klebsiella pneumonia. The U.S. FDA is also currently reviewing the company’s NDA under priority review, with an expected action date in December, 2014.

BMS’ Hep C Treatment Approved By EC

Bristol-Myers Squibb’s Daklinza was approved by the European Commission (EC) this week in combination with other medicinal products indicated for hepatitis C genotypes 1,2,3, and 4. Daklinza, a potent, pan-genotypic NS5A replication complex inhibitor, was combined with sofosbuvir in clinical trials and demonstrated up to 100 percent cure rates in clinical trials. This drug is the first NS5A complex inhibitor approved in the EU and will offer patients a shorter treatment duration of 12 to 24 weeks, as opposed to 48 weeks — the standard length of time following treatment with interferon-and ribavirin-based regimens.

EC Awards Orphan Drug Status To Innate Pharma CTCL Candidate

Innate Pharma’s IPH4102 was awarded orphan drug designation by the EC for the treatment of cutaneous T-cell lymphoma (CTCL). IPH4102 is a first-in-class cytotoxic anti-KIR3DL2 antibody, which is designed to attack and destroy CTCL cancer cells. The drug will be investigated in a Phase 1 clinical trial starting in 2015.

Health Canada Gives Thumbs Up To Eloctate

Biogen Idec announced that Health Canada has approved its Eloctate [Antihemophilic Factor (Recombinant BDD), Fc Fusion Protein] for those suffering from hemophilia A. The drug is indicated to control and prevent bleeding episodes in adults and children, age 12 and up. This is the only therapy approved that effectively treats and reduces bleeding episodes with a prophylactic treatment regimen of every three to five days, or once weekly via infusion. The drug demonstrated its efficacy in the phase 3a LONG study enrolling 165 previously treated males from 12 to 65 years of age.

PNH Drug Granted Orphan Drug Designation In EU

Penn Medicine and Amyndas received orphan drug status for the compound AMY-101 for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The disease, which results in the destruction of red blood cells and thrombosis, causes anemia, and is currently only treatable with a high-priced drug sold in the U.S. The drug aims to inhibit C3, a player in the part of the innate immune system known as “complement” which contains more than 50 proteins in the blood and on cell surfaces and circulates through the body. However, this system can also be inappropriately activated and attack cells, causing inflammatory, immune, and other diseases. The research team examined the role of AMY-101 in cases of self-attack and determined it to be effective.