Top 3 new drug approvals

FDA recently cleared several important new therapies, including the first anticoagulant for children, an injectable for diabetic retinopathy, and a treatment for renal cell carcinoma.

Here are the top 3 new drug approvals:

1.Dalteparin sodium injection (Fragmin, Pfizer) reduces the recurrence of symptomatic venous thromboembolism (VTE) in pediatric patients 1month of age and older.

Fragmin, a type of heparin, was initially approved by FDA in 1994 for adults.

“Prior to this approval, there had been no FDA-approved therapies to treat VTE in pediatric patients. Given the unmet need, we granted the Fragmin application priority review and . . . approving it as the first anticoagulant (blood thinner) indicated for pediatric patients,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA’s Center for Drug Evaluation and Research, in a statement from the agency.

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The efficacy of Fragmin in children was based on a single trial with 38 pediatric patients with symptomatic deep vein thrombosis and/or pulmonary embolism. Twenty-one patients achieved resolution of the qualifying VTE, 7 patients showed regression, 2 patients showed no change, and no patients experienced progression of the VTE, and one patient experienced recurrence of VTE.

2.Aflibercept (Eylea, Regeneron) treats all stages of diabetic retinopathy (DR), reducing the risk of blindness.

Approximately 8 million people live with DR, a complication of diabetes characterized by damage to the blood vessels in the retina.

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The PANORAMA trial showed that by one year, 20% of untreated patients developed proliferative diabetic eye disease, and Eylea reduced this risk by 85% to 88% when administered using an every 16-week or 8-week dosing regimen, respectively, said George D. Yancopoulos, MD, PhD, president and chief scientific officer at Regeneron, in the statement. "In fact, 80% of patients who received the Eylea 8-week dosing regimen had significant improvement in their diabetic retinopathy,” he added.

3.Avelumab (Bavencio, EMD Serono) in combination with axitinib (Inlyta, Pfizer) is the first FDA approval of an anti-PD-L1 therapy as part of a combination regimen for patients with advanced renal cell carcinoma (RCC), EMD Serono and Pfizer said in a statement.

The approval the new combination treatment was based on positive results from a phase 3 study, which found that the combination significantly improved median progression-free survival (PFS) compared with sunitinib (Sutent, Pfizer) by more than five months in the intent-to-treat (ITT) patient population.

“As we look to continue to improve outcomes for people with advanced RCC, new treatment approaches have the potential to benefit patients,” said Robert J. Motzer, MD, Jack and Dorothy Byrne Chair in Clinical Oncology at the Memorial Sloan Kettering Cancer Center and principal investigator for the phase 3 study, in the statement. “With today’s FDA approval of avelumab in combination with axitinib, we can now offer patients with advanced RCC a first-line treatment option that combines a PD-L1 immunotherapy with a well-known VEGFR TKI to provide a significant reduction in the risk of disease progression or death and doubling of the response rate compared with sunitinib.