FDA Action Alert: Dynavax, Ascendis and BMS

FDA Action Alert_Taylor Tieden for BioSpace

Pictured: A scientist with pill bottles in front of FDA headquarters/Taylor Tieden for BioSpace 

The remainder of May will be relatively slow for the FDA, with only four big target action dates. Among other announcements, the regulator is scheduled to release its verdicts on a hepatitis B vaccine for adults on hemodialysis and two potential new indications for BMS's CAR-T therapy Breyanzi. 

Read below for more.

Dynavax Proposes Hepatitis B Vaccine for Patients on Hemodialysis

Today is the day by which the FDA is scheduled to publish its decision on Dynavax Technologies’ sBLA seeking to expand the label of its hepatitis B vaccine Heplisav-B for use in adults on hemodialysis.

Heplisav-B won its initial U.S. approval in 2017 for the prevention of hepatitis B, caused by all known subtypes of the virus, in adults aged 18 years and above. According to its label, Heplisav is a recombinant protein vaccine comprised mainly of the hepatitis B surface antigen, which can prime the body to protect against the virus’ infection.

Heplisav-B is designed to be administered in two doses, and is the only approved hepatitis B shot that completes the vaccination series in one month. The U.K.’s Medicines and Healthcare products Regulatory Agency granted Heplisav-B Marketing Authorization in February 2023.

Dynavax reported that Heplisav-B brought in $213 million in net revenues in 2023, representing a 69% year-over-year increase. As of the end of December 2023, Heplisav-B controlled approximately 42% of the U.S. hepatitis B vaccine market, up from 35% at the end of 2022.

In a statement accompanying the company’s full-year results, CEO Ryan Spencer said that Heplisav-B saw “record revenue growth” in 2023 and became the “market share leader in two largest growth segments.” These achievements will help Dynavax establish Hepliav-B as “the leading vaccine in the U.S. adult hepatitis B vaccine market,” he said.

The company expects continued growth for Heplisav-B in 2024, anchored by the upcoming potential approval. Dynavax forecasts net product revenues of $265 million to $280 million for Heplisav-B this year.

Ascendis Tries Again for Hypoparathyroidism Approval for TransCon PTH

The FDA is scheduled on May 14 to release its verdict on Ascendis Pharma’s resubmission for its investigational drug TransCon PTH (palopegteriparatide), which is being proposed as a treatment for adults with hypoparathyroidism.

Given by subcutaneous injection once daily and formulated to be long-acting, TransConPTH is a prodrug of the parathyroid hormone that works by restoring physiologic levels of the hormone for 24 hours each day. This mechanism of action could potentially allow it to counter hypoparathyroidism, a rare disorder characterized by the insufficiency of parathyroid hormone, in turn leading to low calcium and high phosphate levels.

Patients with hypoparathyroidism experience weakness, muscle cramps and headaches, and the condition can lead to long-term complications such as calcium deposits in the eyes, brain and kidneys. Currently, hypoparathyroidism is managed through high-dose therapy with calcium and active vitamin D, which does not optimally control the condition.

Ascendis first submitted a drug application for TransCon PTH in 2022, which the FDA rejected in May 2023, citing manufacturing concerns. The regulator did not identify problems with the drug’s safety or efficacy, and it did not ask for additional studies.

Currently, TransCon PTH is approved in the U.K. and European Union, where it is marketed under the brand name Yorvipath.

BMS Awaits Two Verdicts for CAR-T Therapy Breyanzi

To close out the month, BMS is looking at two target action dates for its CAR-T therapy Breyanzi (lisocabtagene maraleucel): May 23 for relapsed or refractory follicular lymphoma (FL) and May 31 for relapsed or refractory mantle cell lymphoma (MCL).

In FL, Breyanzi is backed by the Phase II single-arm TRASCEND FL study, which, with 213 enrolled patients, is the largest clinical trial so far to assess a CAR T therapy in relapsed or refractory indolent non-Hodgkin lymphoma, including FL.

BMS presented data from TRANSCEND FL in December 2023, showing that Breyanzi elicited a 95.7% complete response rate in patients with high-risk relapsed or refractory FL when used in the second-line setting. Median progression-free survival had not been reached at the time.

Meanwhile, BMS is backing Breyanzi’s MCL application with findings from the MCL cohort of the TRANSCEND NHL 001 study. Published in December 2023 in the Journal of Clinical Oncology, results from the pivotal Phase I trial demonstrated significant and clinically meaningful treatment responses in heavily pre-treated patients, most of whom achieved a complete response.

Breyanzi is a CAR-T cell therapy that works by targeting the CD19 surface protein, which is commonly expressed on B cells. Once bound to CD19, Breyanzi proliferates, induces the release of proinflammatory cytokines and cell death in cancer cells. The FDA first approved Breyanzi in February 2021 for relapsed or refractory B-cell lymphoma.

In March 2024, Breyanzi picked up two new approvals, one for chronic lymphocytic leukemia and the other for small lymphocytic leukemia.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Correction (May 14): This story has been updated from its original version to correct that TransConPTH is given by subcutaneous injection as opposed to orally. BioSpace regrets the error. 

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