News Feature | July 14, 2014

What You Missed From The FDA Last Week July 7-11, 2014

By Anna Rose Welch

Last week, the FDA announced its decisions about drugs from Eagle Pharma, Galderma, and Boehringer Ingelheim, among others. Also in the news, collaborative molecular screening platforms could be the answer for companies seeking new cancer candidates, and global drug discovery outsourcing is on the rise.

Beleodaq Receives FDA Approval For PTCL

Last Thursday, the FDA announced that it approved via the accelerated approval program Spectrum Pharma’s Beleodaq (belinostat) for patients suffering from peripheral T-cell lymphoma (PTCL). Beleodaq is the third drug approved to treat this form of Hodgkin’s lymphoma, following the approvals of Folotun (pralatrexate) and Istodax (romidepsin) in 2009 and 2011 respectively. Beleodaq is a pan-histone deacetylase (HDAC) inhibitor which obstructs three classes of zinc-dependent HDAC enzymes, in turn influencing chromatin accessibility, gene transcription, and clinical activity in cancer patients.

Recordati Awarded Orphan Drug Designation For Organic Acidemias

Recordati’s Carbaglu has been given orphan drug designation to be used in the treatment of organic acidemias (OAs). Several common OAs include isovaeric academia, propionic academia, and methylmalonic academia, through which the the body loses non-amino organic acids through the urine. Recordati currently markets Carbaglu for the treatment of acute hyperammonemia due to hepatic N-acetylglutamate synthase deficiency.

Eagle Pharma Injection Solution Receives Orphan Drug Designation

The FDA granted Orphan Drug Designation to Eagle Pharmaceuticals’ bendamustine hydrochloride (HCl) injection solution for chronic lymphocytic leukemia (CLL) and indolent B-cell non-Hodgkins lymphoma. The product is diluted with 50mL saline and administered via infusion. The infusion lasts roughly 10 minutes, making this product a direct competitor against Treanda from Cephalon (subsidiary of Teva), which requires longer dilution times (30-60 mins).

Akashi Therapeutics Receives Fast-Track Designation For HT-100

Akashi Therapeutics announced that its HT-100 (delayed-release halofuginone) candidate for boys with Duchenne Muscular Dystrophy (DMD) has received fast track designation. The small molecule candidate, which is currently in a phase 1b/2a multicenter clinical trial, aims to reduce inflammation and fibrosis and encourage muscular regeneration in boys with DMD. 

Galderma’s Congenital Ichthyosis Drug Given Orphan Drug Designation

Galderma announced that its trifarotene molecule has been granted Orphan Drug Designation for the treatment of congenital ichthyosis — including Lamellar ichthyosis. The molecule, which is being examined as a treatment for various skin diseases because of its retinoid functionality and keratolytic properties, will be the focus of a new clinical development plan.

Boehringer Ingelheim’s Nintedanib Granted Priority Review

The FDA has accepted Boehringer Ingelheim’s New Drug Application (NDA) for its idiopathic pulmonary fibrosis (IPF) treatment, nintedanib (small molecule tyrosine kinase inhibitor). In its application, the company has included results from two global phase 3 trials measuring the safety and efficacy of the treatment in this rare and deadly lung disease. This drug stands to meet an urgent unmet medical need, as roughly 132,000 Americans suffer from IPF, which currently has no FDA-approved treatment options. Nintedanib was given orphan drug designation in 2011.

FDA Grants CASI Pharma’s ENMD-2076 Orphan Drug Designation

CASI Pharmaceutical’s Aurora A/angiogenic kinase inhibitor, ENMD-2076, was given orphan drug designation for the treatment of hepatocellular carcinoma (HCC). ENMD-2076 targets both the VEGFR, Flt-3 and FGFR3 kinases, along with the Aurora A kinase implicated in several cancers. The drug has been tested on ovarian, breast, liver, renal, and sarcoma cancers in phase 1 clinical trials. ENMD-2076 is currently in Phase 2 trials for the treatment of ovarian cancer and triple negative breast cancer, among others. The drug was previous granted orphan drug designation for ovarian cancer, multiple myeloma, and acute myeloid leukemia.

Pharma Talking Points

In The Biosimilarz Blog last week, Dr. Duncan Emerton provides his take on the recent EU positive opinion of Eli Lilly and partner Boehringer Ingelheim’s biosimilar Abasria in Europe. This move, Emerton argues, is a significant move for the industry for several key reasons, including the fact that this may be the first biosimilar insulin to be approved anywhere in the world. He also says that this move will help balance out Lilly’s portfolio significantly, considering the fact that the company has yet to offer a long-acting modern insulin. He also discusses a few key questions surrounding this new development, including Novo’s and Sanofi’s response to the new competition, future contenders for insulin biosimilars, and how this product will be priced.

The European Organization for Research and Treatment of Cancer (EORTC) has published a paper arguing that collaborative molecular screening platforms are necessary in identifying new cancer drug candidates. The article can be found in the June 17, 2014 edition of Nature Reviews Clinical Oncology.

A coalition has approached the FDA with a letter requesting the FDA avoid giving biosimilars unique names, the Wall Street Journal reports. Thirty-two pharmacies, health insurers, unions, and pension plans voiced their concerns in this July 1st letter. According to the coalition, if drugs were to have different International Nonproprietary Names (INN), there would be increased patient/prescriber confusion, medication errors, and cause a disconnect from the molecule’s safety information.

A Life Science Leader article by Jim Zhang, president of JZMed, discusses the rise in global drug discovery outsourcing — the market for which is forecasted to hit $25B by 2018, Zhang says. The article discusses the effects of the new drug discovery and outsourcing strategies that are currently evolving in the global pharma/biopharm space. Zhang also discusses the important of emerging countries in small molecule drug discovery. 

A PMLive article highlights some of the ways Big Pharma is approaching the orphan drug sector —  methods which ultimately suggests that a commercial model is emerging within the industry. Indeed, orphan drugs could be the answer for companies looking to improve profits and see increased demand. Partnerships between scientists and regulators, collaboration with academic research units, and building resources, are some of the ways pharma companies are beginning to crack into this difficult market.