FDA News – May 2015

May 2015, Vol 6, No 4 - FDA Approvals, News & Updates


Venetoclax Receives FDA Breakthrough Therapy Designation for Relapsed or Refractory CLL and 17p Deletion

The FDA granted venetoclax (RG7601, GDC-0199/ABT-199; Roche/AbbVie) a breakthrough therapy designation for the treatment of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) and the 17p deletion genetic abnormality. Venetoclax, an investigational small-­molecule inhibitor of the BCL-2 protein, an apoptosis regulator, is being investigated for several hematologic cancers in addition to CLL.

A breakthrough therapy designation allows the drug manufacturer to accelerate the development of the drug, as well as guarantees the acceleration of the FDA review process for medications that are intended to treat life-­threatening diseases, when the evidence shows that the specific medication may provide a substantial improvement to patients compared with currently available therapies on the market.

“People who have relapsed or refractory CLL with a 17p deletion typically have a poor prognosis, and do not respond to many currently available treatment options,” said Sandra Horning, MD, Chief Medical Officer and Head of Global Clinical Development at Roche. “We are pleased that the FDA has granted venetoclax Breakthrough Therapy designation and hope this regulatory pathway will help us bring venetoclax to people with this difficult-to-treat disease soon,” she explained.

Approximately 30% to 50% of patients with relapsed or refractory CLL have the 17p deletion; these patients have poor results with conventional chemotherapy, with a median life expectancy of <3 years.

(May 7, 2015)




FDA Grants Orphan Drug Status to Cantrixil, an Investigational Chemotherapy for Ovarian Cancer

The FDA has granted orphan drug designation to Cantrixil (Novogen/CanTx) for the treatment of patients with ovarian cancer. Cantrixil is a cyclodextrin envelope that contains the active ingredient TRXE-002. Cantrixil is designed as an intracavity chemotherapy intended for injection into the peritoneal and pleural cavities, without any local irritation or toxicity. The goal of using this construct is to reach high drug levels in the area where cancer cells spread.

The FDA granted this designation to cantrixil after reviewing preliminary preclinical data from the drug manufacturer.

Graham Kelly, PhD, Novogen and CanTx Chief Executive Officer, said, “Receiving this designation is one more step in our objective of bringing Cantrixil to market as a drug that we hope will provide meaningful clinical benefit to patients with ovarian cancer and deliver that long-sought breakthrough for patients with a cancer that has shown only slight improvement in 5-year survival rates over the last 30 years.”

Dr Kelly added, “CanTx came out of a belief by Yale University and some long-term ovarian cancer researchers in the Yale Medical School that Cantrixil represented a potential breakthrough in the treatment of ovarian cancer.” (CanTx is a venture company that was established by researchers at Yale University.)

Orphan drug designation for an investigational drug is intended to promote drug development for rare diseases with small patient populations. This designation can provide the drug manufacturer several incentives to encourage and to support the development of drugs with low incidence rates, including financial subsidies for clinical research, tax incentives, extended regulatory patent protection, and enhanced marketing rights upon approval of the drug.

Preclinical data for Cantrixil were presented by the Yale researchers at the recent 2015 Annual Conference of the American Association for Cancer Research. That data represented the key criteria that the researchers had set for Cantrixil to justify its transition to clinical trials. The data indicate that Cantrixil in patients with late-stage chemoresistant ovarian cancer can effect a very significant (>95%) tumor reduction.

Cantrixil is expected to be available for use in Australia (Novogen is an Australian company) in late 2015 or early 2016 for patients with malignant ascites, a terminal condition associated with ovarian cancer and other types of cancer for which no other therapy is available; this is being recognized as the first step before the regulatory approval of this agent for ovarian cancer in the United States or in Australia. Researchers will ultimately seek a primary indication for Cantrixil as first-line therapy for early-stage cancers of the abdominal cavity, such as ovarian, uterine, colo­rectal, and gastric carcinomas.

Approximately 85% of advanced cases of ovarian cancer respond to first-line chemotherapy, but the majority of these cases relapse within several years.

(April 21, 2015)