FDA Action Alert: Omeros, Oyster Point, BeiGene and Ocular

FDA_Sarah Silbiger/Getty Images

Sarah Silbiger/Getty Images

The U.S. Food and Drug Administration (FDA) has a few PDUFA dates on its calendar for this week. Here’s a look.

Omeros’ Narsoplimab for Hematopoietic Stem Cell Transplant-Associated TMA

Omeros Corporation has a target action date of October 17, 2021, for its Biologics License Application (BLA) for narsoplimab for treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). This date is a three-month extension after the agency requested more information, which the company did. The FDA then declared it a major amendment and required three more months to review.

On October 1, the FDA notified the company that as part of its review of the BLA it had identified deficiencies that “preclude discussion of labeling and post-marketing requirements/commitments at this time.” As a result, the company did not expect to resolve the issues with the agency by October 17.

Narsoplimab is the first drug candidate the FDA has received for approval in HSCT-TMA. The drug has Breakthrough Therapy and Orphan designations for HSCT-TMA and IgA nephropathy. HSCT-TMA is a severe complication of HSCT and can affect 20% to 30% of recipients. It can lead to end-organ injury and poor outcomes, particularly involving the kidney.

Oyster Point’s Varenicline for Dry Eye Disease

Oyster Point Pharma has a target action date of October 17 for its New Drug Application for varenicline nasal spray for dry eye disease. The NDA was built on results from the Phase III ONSET-2, Phase IIb ONSET-1 and Phase II MYSTIC trials in more than 1,000 patients with mild, moderate or severe symptoms of dry eye disease. In those studies, the drug demonstrated statistically significant improvements compared to control.

At the company’s second-quarter financial report on August 25, Jeffrey Nau, President and Chief Executive Officer of Oyster Point said they were preparing for the potential launch of varenicline in the fourth quarter. “We are also excited to be building our commercial field force and capabilities as we welcome our first sales representatives to Oyster point Pharma in July, with our mission to provide a novel therapeutic treatment for dry eye disease patients in Q4’21, if approved by the FDA.”

BeiGene’s Brukinsa for Waldenstrom’s Macroglobulinemia

BeiGene had a target action date of October 18 for its supplemental new drug application (sNDA) for Brukinsa (zanubrutinib) for adults with Waldenstrom’s Macroglobulinemia (WM). The FDA approved it for this indication on September 1. 

“We are delighted by today’s FDA approval for Brukinsa in its second indication, offering a new treatment option with demonstrated efficacy and safety benefits for patients with Waldenstrom’s macroglobulinemia,” said Jane Huang, Chief Medical Officer, Hematology at BeiGene.

The submission included data from 351 patients with WM and was built primarily on data from the Phase III ASPEN study of zanubrutinib compared to ibrutinib for WM, with supportive data from the Phase II trial of zanubrutinib in relapsed/refractory WM that was conducted in China and the Phase I/II trial in patients with B-cell malignancies.

The drug received accelerated approval in the U.S. in November 2019 for mantle cell lymphoma in adults with at least one previous therapy. The drug is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK).

Ocular’s Dextenza for Extracanalicular Use

Ocular Therapeutix had a target action date of October 18 for its sNDA for Dextenza (dexamethasone ophthalmic insert) 0.4 mg for intracanalicular use. It would include treatment of ocular itching associated with allergic conjunctivitis.

The drug is approved for ocular inflammation and pain after ophthalmic surgery. It is a corticosteroid intracanalicular insert placed in the punctum, which is a natural opening in the inner part of the lower eyelid, and into the canaliculus. It is designed to deliver dexamethasone to the ocular surface for up to 30 days without preservatives.

The safety of the drug was evaluated in three Phase III trials and a Phase II trial.

On October 11, the FDA approved the sNDA for this indication.

“Allergic conjunctivitis is a common condition seen in the offices of eye care providers,” said Michael Goldstein, President, Ophthalmology and Chief Medical Officer of Ocular Therapeutix. “We are really excited about this label expansion and the potential benefits for patients. The use of topical steroids is an important part of our current clinical armamentarium in the treatment of allergic conjunctivitis. Dextenza can now provide an office-based, physician-administered preservative-free method of steroid delivery that benefits patients with ocular itching associated with allergic conjunctivitis.”

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