AMAG Pharmaceuticals achieves key milestones in next generation development programs

The definitive pharmacokinetic study and the comparative pain study for the Makena (hydroxyprogesterone caproate injection) subcutaneous auto-injector program (Makena SQ) have been initiated with the first patient dosed.

The enrollment of patients in the Feraheme Phase 3 label expansion trial for the treatment of iron deficiency anemia (IDA) has surpassed approximately two-thirds of the total target enrollment of 2,000 patients, resulting in the acceleration of the estimated supplemental new drug application (sNDA) filing date to mid-2017. 

AMAG chief executive officer William Heiden said: “We are pleased to announce significant progress on these two next generation development programs.

“The Makena subcutaneous auto-injector is designed for easier administration by healthcare providers and potentially less painful injections for patients, and we are focused on completing the development of this product and bringing it to market as quickly as possible.

"With an earlier sNDA filing for Feraheme, we hope to receive FDA approval for a broader label sooner than previously forecasted, which if approved, could significantly expand the number of patients who could benefit from Feraheme therapy in the future.”

Makena subcutaneous auto-injector development program

The definitive pharmacokinetic study for the Makena SQ program is a randomized open label parallel study designed to demonstrate comparable bioavailability between subcutaneous and intramuscular injections of Makena. The study is expected to enroll 120 healthy post-menopausal women with a 1:1 randomization.

The comparative pain study is also a randomized open label parallel study designed to compare the average injection pain of four weekly injections between the subcutaneous and intramuscular injections of Makena using a validated pain scale.

The study is expected to enroll 60 healthy, post-menopausal women with a 1:1 randomization. The company intends to use the results from the pain study to establish clinical superiority of the SQ auto-injector over the intramuscular injection to support a submission to the FDA for orphan drug exclusivity for the Makena SQ auto-injector.

AMAG plans to file the sNDA for the Makena SQ auto-injector in the second quarter of 2017 and anticipates a decision on approval in the first quarter of 2018.

Phase 3 clinical trial evaluating the safety of Feraheme compared to Injectafer

This is the final trial that the company plans to conduct in preparation for filing the sNDA to broaden the use of Feraheme beyond the current chronic kidney disease (CKD) indication to include all adult IDA patients who have failed or cannot tolerate oral iron treatment or in whom oral iron was contraindicated.

Patient enrollment in the clinical trial has surpassed two-thirds of the total target enrollment mark ahead of schedule. Based on the rapid pace of enrollment, AMAG now expects to file the sNDA in mid-2017, with a decision on approval expected in the first half of 2018. Approval of the broader IDA label would double the addressable patient population eligible for Feraheme.

The non-inferiority, randomized, double-blind, multicenter clinical trial is evaluating the incidence of moderate-to-severe hypersensitivity reactions (including anaphylaxis), and moderate-to-severe hypotension with Feraheme compared to Injectafer in adults with IDA.

A total of approximately 2,000 patients will be randomized in a 1:1 ratio into one of two treatment groups—Feraheme IV infusion or Injectafer IV infusion. While the trial’s primary endpoint is safety, the trial will also assess efficacy.

AMAG chief medical officer and clinical development and regulatory affairs senior vice president Dr. Julie Krop said: “This clinical trial is progressing well and if the current pace of enrollment continues, we believe that the trial could be completed in early 2017.

“We are grateful to the investigators participating in the trial whose enthusiasm and commitment have allowed us to enroll the trial much faster than anticipated, potentially allowing a broader population of patients suffering from IDA to benefit from Feraheme earlier than previously expected.

"We are also pleased that the Makena next generation subcutaneous auto-injector program is progressing well and we remain on track for our sNDA filing next year.”