GlaxoSmithKline’s promising new blood cancer drug has received another big boost, this time being granted “breakthrough” status by the Food and Drug Administration.

The FDA and GSK announced the designation Thursday.

Just a month ago, a similar status was awarded to the drug candidate by European drug authorities as well as an “orphan” designation.

[What do designations mean? See details below.]

GSK2857916 is the title of the drug, which is still under development in clinical trials. It targets multiple myeloma.

It is described by GSK as “an anti B-cell maturation agent (BCMA) monoclonal antibody-drug conjugate.”

“Oncology R&D at GSK is focussed on developing medicines with transformational potential for patients and we are pleased that our investigational antibody-drug conjugate is the first BCMA targeting agent to receive Breakthrough Therapy and PRIME designation,” said Axel Hoos, who is senior vice president for oncology research and development at GSK.

PRIME refers to the designation for the drug awarded by the European Medicines Agency.

“GSK plans to rapidly advance clinical trials with this promising therapy, alone and in combination with other therapies, to further investigate how GSK2857916 could benefit patients with multiple myeloma,” Hoos added. “The monotherapy data that we have seen for GSK2857916 support its transformational potential and we look forward to working with regulators as we progress the development program.”

GSK, which maintains an R&D operation in RTP and a manufacturing plant in Zebulon, sold its cancer drug portfolio to Novartis in 2015 but continues R&D efforts.

The designations come as GSK prepares to announce trial results in December.

What do designations mean?

Here’s how GSK explains what the designations mean:

  • Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s). Drugs that receive breakthrough therapy designation are eligible for all features of FDA’s Fast Track Program.
  • PRIME designation is offered by the European Medicines Agency (EMA) to enhance support for the development of medicines that target an unmet medical need. It is based on enhanced interaction between sponsor companies and the EMA to optimize development plans and speed up evaluation so these medicines can reach patients earlier.
  • Orphan designation may be granted for therapies intended to treat conditions that affect fewer than 200,000 people in the U.S. The designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including tax credits for qualified clinical testing

Source: GSK