Bristol Myers Squibb Announces New Prescription Drug User Fee Act Deadline for Supplemental Biologics License Application for Reblozyl® (luspatercept-aamt)

PRINCETON, New Jersey–(BUSINESS WIRE)–Bristol-Myers Squibb (NYSE:BMY) today announced that the U.S. Food and Drug Administration (FDA) has extended review of the supplemental license application of biological products (sBLA) for Reblozyl® (luspatercept-aamt) for the treatment of anemia in adults with non-transfusion dependent beta-thalassemia (ATN) through June 27, 2022. Reblozyl is developed and marketed through a global collaboration with Merck & Co., Inc., known as MSD outside the United States and Canada, following Merck’s acquisition of Acceleron Pharma, Inc. . in November 2021.

A written response to a request for information has been determined by the FDA to constitute a major amendment; therefore, the Agency has extended the Prescription Drug User Fee Act (PDUFA) deadline by three months to allow time for a full review of the submission. The sBLA was based on safety and efficacy results from the pivotal Phase 2 BEYOND study evaluating Reblozyl as well as the best supportive care for adults with beta-thalassemia NTD. An application (type II variation) is also under review by the European Medicines Agency.

Reblozyla first-in-class treatment option, is currently approved in the United States and the European Union to treat beta-thalassemia associated with transfusion-dependent anemia and low-risk myelodysplastic syndromes in the event of agent failure stimulating erythropoietin (ESA) and requiring red blood cell transfusions.

On Reblozyl®

Reblozyl, a first-rate therapeutic option, promotes late maturation of red blood cells in animal models.1 Reblozyl is being developed and commercialized through a global collaboration with Merck following Merck’s acquisition of Acceleron Pharma, Inc. in November 2021. Reblozyl is currently approved in the United States for the treatment of:

  • anemia in adult patients with beta-thalassemia who require regular red blood cell transfusions, and
  • anemia in failure of an erythropoiesis-stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low-to-intermediate risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or d a myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

Reblozyl is not indicated as a substitute for red blood cell transfusions in patients requiring immediate correction of anemia.

Important Safety Information

WARNINGS AND PRECAUTIONS

Thrombosis/Thromboembolism

In adult patients with beta-thalassemia, thromboembolic events (TEEs) were reported in 8/223 (3.6%) patients treated with REBLOZYL. TEEs included deep vein thrombosis, pulmonary embolism, portal vein thrombosis, and ischemic stroke. Patients with known risk factors for thromboembolism (splenectomy or concurrent use of hormone replacement therapy) may be at increased risk for thromboembolic disease. Consider thromboprophylaxis in patients at increased risk of TEE. Monitor patients for signs and symptoms of thromboembolic events and institute treatment promptly.

Hypertension

Hypertension was reported in 10.7% (61/571) of patients treated with REBLOZYL. In clinical studies, the incidence of grade 3 to 4 hypertension ranged from 1.8% to 8.6%. In beta-thalassemia patients with normal baseline blood pressure, 13 (6.2%) patients developed systolic blood pressure (SBP) ≥ 130 mm Hg and 33 (16.6%) patients developed blood pressure (DBP) ≥ 80 mm Hg. In adult MDS patients with normal baseline blood pressure, 26 (29.9%) patients developed DBP ≥ 130 mm Hg and 23 (16.4%) patients developed a DBP ≥ 80 mm Hg. Monitor blood pressure before each administration. Manage new or exacerbations of pre-existing hypertension with antihypertensive agents.

Embryo-foetal toxicity

REBLOZYL can cause fetal harm when administered to a pregnant woman. REBLOZYL caused increased post-implantation loss, decreased litter size, and increased incidence of skeletal variations in studies in pregnant rats and rabbits. Inform pregnant women of the potential risk to the fetus. Advise women of childbearing potential to use effective contraception during treatment and for at least 3 months after the last dose.

SIDE EFFECTS

Beta Thalassemia

  • Serious adverse reactions occurred in 3.6% of patients taking REBLOZYL. Serious adverse reactions occurring in 1% of patients included stroke and deep vein thrombosis. A fatal adverse reaction occurred in 1 patient treated with REBLOZYL who died following an unconfirmed case of acute myeloid leukemia (AML).
  • The most common adverse reactions (at least 10% for REBLOZYL and 1% more than for placebo) were headache (26% versus 24%), bone pain (20% versus 8%), arthralgia ( 19% vs 12%), fatigue (14% vs 13%), cough (14% vs 11%), abdominal pain (14% vs 12%), diarrhea (12% vs 10%) and dizziness (11% vs 5%).

Myelodysplastic syndromes

  • To note >3 (≥2%) adverse reactions included fatigue, hypertension, syncope, and musculoskeletal pain. A fatal adverse reaction occurred in 5 (2.1%) patients.
  • The most common adverse reactions (≥10%) were fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, respiratory tract infections upper abdomen, bronchitis and urinary tract infections.

LACTATION

It is not known whether REBLOZYL is excreted in breast milk or absorbed systemically after ingestion by an infant. REBLOZYL has been detected in the milk of lactating rats. When a drug is present in animal milk, it is likely that the drug will be present in human milk. Because many drugs are excreted in human milk and because of the unknown effects of REBLOZYL in infants, a decision must be made whether to discontinue breast-feeding or to discontinue treatment. Due to the risk of serious adverse reactions in the breast-fed child, breast-feeding is not recommended during treatment and for 3 months after the last dose..

Please see the complete prescribing information for REBLOZYL.

Bristol Myers Squibb: creating a better future for people with cancer

Bristol Myers Squibb is inspired by a single vision: to transform people’s lives through science. The company’s cancer research goal is to provide medicines that give every patient a better, healthier life and make a cure a possibility. Building on a legacy across a wide range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine and, through innovative digital platforms, transforming data into information that sharpens their concentration. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to examine cancer from all angles. Cancer can have an unrelenting grip on many aspects of a patient’s life, and Bristol Myers Squibb is committed to taking steps to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb strives to empower all people with cancer for a brighter future.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients overcome serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, TwitterYoutube, Facebook and Instagram.

Juno Therapeutics, Inc. is a wholly owned subsidiary of Bristol-Myers Squibb Company. In certain countries outside of the United States, due to local laws, Celgene and Juno Therapeutics are referred to as Celgene, a Bristol Myers Squibb Company and Juno Therapeutics, a Bristol Myers Squibb Company.

Bristol Myers Squibb Caution Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 relating to, among other things, the research, development and marketing of pharmaceuticals. All statements that are not statements of historical fact are, or may be deemed to be, forward-looking statements. These forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, deflect or modify any of them in the next few years. years, which are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed or implied by the statements. These risks, assumptions, uncertainties and other factors include, among others, the possibility that Reblozyl (luspatercept-aamt) may not receive regulatory approval for the additional indication described in this release within the currently anticipated time frame or at all, that any marketing authorization, if granted, may have important limitations as to their use and, if approved, whether this product candidate for the additional indication described in this release will be commercially successful. No forward-looking statement can be guaranteed. The forward-looking statements contained in this press release must be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the Cautionary Note and Discussion of Risk Factors in the report. Annual Report of Bristol Myers Squibb on Form 10-K for the year ended December 31, 2021, as updated by our subsequent quarterly reports on Form 10-Q, our current reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and, except as required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether whether as a result of new information, future events, changed circumstances or otherwise.

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The references:

  1. Galanello R, Origa R. Beta thalassemia. Orphanet review of rare diseases. 2010;5(11). Available at: https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-5-11. Accessed March 2022.


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