News Feature | August 4, 2014

What You Missed From The FDA Last Week—July 28th-August 1, 2014

By Anna Rose Welch

FDA Approves Two Boehringer Ingelheim Drugs

Last Thursday and Friday, the FDA approved two new drugs from Boehringer Ingelheim: Striverdi Respimat and Jardiance.

Striverdi Respimat is an inhalation spray indicated for the treatment of COPD, including chronic bronchitis and/or emphysema. The treatment is approved for once daily, long-term use. A long-acting beta-andrenergic agonist (LABA), Striverdi Respimat relaxes airway muscles in the lungs and improves lung function. The drug was tested in 3,104 COPD patients.

Jardiance (empagliflozin) tablets were  approved as a therapy for type 2 diabetes, in addition to diet and exercise. The drug, a sodium glucose co-transporter 2 (SGLT2) inhibitor, aids in glycemic control by blocking glucose reabsorption by the kidney, increasing glucose excretion, and lowering blood glucose levels.  The drug was investigated in seven clinical trials involving 4,480 patients with type 2 diabetes. Patients with type 1 diabetes are not eligible for treatment with Jardiance. The FDA has also required four post-marketing studies for Jardiance.

FDA Approves Expanded Label For Imbruvica

Janssen’s and Pharmacyclics’ Imbruvica was approved for use in patients with chronic lymphocytic leukemia (CLL) who possess a 17p deletion. The drug previously received accelerated approval to treat CLL, however new clinical trial results demonstrate that the drug reduced disease progression in 78 percent of patients and extended overall survival in 57 percent of patients. For those with 17p deletion, 75 percent experienced a reduction in the risk of disease progression or death. The target date for the drug’s prescription drug user fee is October 2014. There are currently ongoing studies examining Imbruvica’s ability to treat patients with mantle cell lymphoma who have received at least one prior therapy.

Eylea Given Thumbs Up For Diabetic Macular Edema

Regeneron Pharma’s Eylea (aflibercept) injection was approved for the treatment of Diabetic Macular Edema (DME). Recommended dosage for the intravitreal injection is 2 mg every two months after 5 initial monthly injections. Data from the one-year, phase 3 VISTA-DME and VIVID-DME studies involving 862 patients supported the approval process. In both studies, patients taking Eylea gained the ability to read roughly two additional lines on an eye chart when compared to those taking placebo.

Fate Therapeutics Receives IND Clearance For Prohema

The FDA cleared Fate Therapeutics’ IND for clinical development of Prohema for pediatric patients undergoing hematopoietic stem cell transplantation in order to treat inherited metabolic disorders (IMDs). With the approval of the IND, the company will now be able to expand its clinical investigation into rare, non-malignant disorders. Enrollment of IMD pediatric patients for the PROVIDE trial will begin in the fourth quarter of 2014.

FDA BPAC Gives Favorable Recommendation Of HyQvia

Baxter and Halozyme Therapeutics announced that the Blood Products Advisory Committee voted 15-1 in favor of HyQvia (Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase). The subcutaneous treatment is for patients with primary immunodeficiency. The drug has been previously approved in Europe in 2013 for adults suffering from myeloma and chronic lymphocytic leukemia and primary immunodeficiency syndromes.

Pharma Talking points

NPR recently published an article discussing a recent workshop in which the FDA discussed the need for a quantitative way of presenting prescribing instructions to patients and physicians. Two panelists from Dartmouth presented a format known as the “drug facts box” that aims to show how the drug compares to the placebo.

According to the Wall Street Journal blog, the FDA has received an unexpectedly large number of applications to market generic drugs. An unnamed expert tells the WSJ that this “backlog reflects a more fundamental problem the FDA is having with the growing amount of paperwork, and this threatens to derail agency goals to reduce outstanding requests to approve generic drugs.” For the current fiscal year, the agency has received 1,440 applications—600 of which came in during June. The FDA acknowledges the large workload but is not concerned. A spokeswoman claimed that the agency expects fewer applications to come in over the next few months and that the agency will continue to use its resources to hire and train personnel as needed.

The Health Affairs Blog features a post on expanded access or “compassionate use” programs. According to the blog, a recent meeting was held in order to identify common themes and outlines to come up with potential industry-wide policies for expanded access. The groups in attendance discussed their experiences in certain clinical cases, identified internal industry-specific structural barriers, and highlighted situations where expanded access would be inappropriate or where regulations or liability concerns could hinder expanded access.