FDA Grants Breakthrough Therapy Designation for Venclexta in Combination With Azacitidine for the Treatment of Patients With Myelodysplastic Syndromes

Every year in the United States, approximately 10,000 people are diagnosed with myelodysplastic syndromes (MDS), and there remains a high unmet need for new treatment options

The designation is based on interim results from the Phase Ib M15-531 study investigating Venclexta plus azacitidine in people with previously untreated higher-risk MDS

This is the 11th Breakthrough Therapy Designation for Genentech’s hematology medicines and the sixth for Venclexta, demonstrating its potential across multiple blood cancers

SOUTH SAN FRANCISCO, CA, USA I July 20, 2021 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that Venclexta® (venetoclax) in combination with azacitidine has been granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with previously untreated intermediate, high- and very high-risk myelodysplastic syndromes (MDS) based on the revised International Prognostic Scoring System (IPSS-R). MDS are a rare group of blood cancers that gradually affect the ability of the bone marrow to produce normal blood cells. This can lead to weakness, frequent infections, anemia and debilitating fatigue that can profoundly affect a person’s quality of life. In some cases, MDS can also progress into acute myeloid leukemia (AML). Every year in the United States, approximately 10,000 people are diagnosed with MDS, and the median survival for those with higher-risk MDS is approximately 18 months.

“Higher-risk MDS is associated with poor prognosis, reduced quality of life, and limited treatment options,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “We are pleased that the FDA has granted Venclexta its sixth Breakthrough Therapy Designation in recognition of its potential to improve outcomes for people with MDS in combination with azacitidine.”

This designation was granted based on interim results from the Phase Ib M15-531 study investigating Venclexta plus azacitidine in people with previously untreated, higher-risk MDS. BTD is designed to accelerate the development and review of medicines intended to treat serious or life-threatening conditions with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies. This is the 38th BTD for Genentech’s portfolio of medicines, and the 11th designation for its hematology portfolio.

This most recent designation reinforces the potential of Venclexta-based combinations across several blood cancers, including MDS. In the United States, Venclexta has been granted six BTDs by the FDA: one for previously untreated chronic lymphocytic leukemia (CLL), two for relapsed or refractory CLL, two for previously untreated AML, and one for MDS. Venclexta is already approved in the United States in combination with azacitidine, decitabine or low-dose cytarabine for the treatment of newly diagnosed AML in adults 75 years or older, or who have comorbidities that preclude use of intensive induction chemotherapy, and in the European Union in combination with hypomethylating agents, azacitidine and decitabine, for the treatment of adult patients with newly diagnosed AML who are ineligible for intensive chemotherapy. Venclexta is also approved in the United States and European Union in combination with Rituxan® (rituximab) for the treatment of adult patients with CLL who have received at least one prior therapy; in combination with Gazyva® (obinutuzumab) for the treatment of adult patients with previously untreated CLL; and as a monotherapy for the treatment of CLL in the presence of 17p deletion or TP53 mutation in people who are unsuitable for or have failed a B-cell receptor pathway inhibitor.

Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States.

About Myelodysplastic Syndromes (MDS)
MDS are a rare group of blood cancers that gradually affect the ability of the bone marrow to produce normal blood cells. This can lead to weakness, frequent infections, anemia and debilitating fatigue. In some cases, MDS can also progress into acute myeloid leukemia (AML). Every year in the United States, approximately 10,000 people are diagnosed with MDS, and the median survival for those with higher-risk MDS is approximately 18 months.

There are several classifications of MDS – very low-risk to very high-risk – determined by the composition of the bone marrow, blood cell counts, and chromosomal alterations. Higher-risk disease is defined as intermediate, high- or very high-risk based on the revised International Prognostic Scoring System (IPSS-R), which is a risk assessment scale that uses five prognostic indicators to predict the course of a patient’s disease. Approximately half (45%) of patients present with higher-risk MDS, which is associated with a poorer prognosis and short life expectancy.

About the M15-531 study
The M15-531 [NCT02942290] study is a Phase Ib, open-label, non-randomized, multicenter, dose-finding study evaluating Venclexta® (venetoclax) in combination with azacitidine in treatment-naïve patients with higher-risk myelodysplastic syndromes (MDS) comprising a dose-escalation portion and a safety expansion portion. The primary objectives of the study are to assess the safety profile and pharmacokinetics and determine the recommended Phase II dose and dosing schedule of Venclexta in combination with azacitidine. The response criteria specified in the M15-531 study are based on the modified International Working Group 2006 response criteria for MDS.

About Venclexta
Venclexta is a first-in-class targeted medicine designed to selectively bind and inhibit the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers and other tumors, BCL-2 builds up and prevents cancer cells from dying or self-destructing, a process called apoptosis. Venclexta blocks the BCL-2 protein and works to help restore the process of apoptosis.

Venclexta is being developed by AbbVie and Genentech, a member of the Roche Group. It is jointly commercialized by the companies in the United States and commercialized by AbbVie outside of the United States. Together, the companies are committed to research with Venclexta, which is currently being studied in clinical trials across several types of blood cancers.

Venclexta Indications
Venclexta is a prescription medicine used:

  • to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
  • in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly-diagnosed acute myeloid leukemia (AML) who:

‒ are 75 years of age or older, or

‒ have other medical conditions that prevent the use of standard chemotherapy.

It is not known if Venclexta is safe and effective in children.

Please see the Venclexta full Prescribing Information,including the Medication Guide,for additional Important Safety Information.

About Genentech in Hematology
For more than 20 years, Genentech has been developing medicines with the goal to redefine treatment in hematology. Today, we’re investing more than ever in our effort to bring innovative treatment options to people with diseases of the blood. For more information visit http://www.gene.com/hematology.

About Genentech
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

SOURCE: Genentech

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