FDA Grants Breakthrough Therapy for Roche’s MDS Treatment

Roche recently announced that FDA granted breakthrough therapy designation for its combination myelodysplastic syndromes (MDS) treatment.

The treatment combination of Venclexta and azacitidine is intended for adult patients with previously untreated, high-and very high-risk MDS. MDS is a rare group of blood cancers that can progress into acute myeloid leukemia (AML).

Nearly 10,000 individuals are diagnosed with MDS annually in the US. The median survival for those with higher-risk MDS is about 18 months. 

“Higher-risk MDS is associated with poor prognosis, reduced quality of life, and limited treatment options,” Levi Garraway, MD, PhD, Roche’s chief medical officer and head of global product development, said in the announcement. 

“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,” Garraway continued. 

FDA based its breakthrough therapy designation on results from the Phase 1b M15-531 study investigating Venclexta plus azacitidine in individuals with previously untreated, higher-risk MDS. 

The designation will boost development and review of the combination treatment and reinforce the potential of treatment across several blood cancers, including MDS. 

FDA has granted Venclexta six breakthrough therapy designations, one for previously untreated chronic lymphocytic leukemia (CLL), two for relapsed or refractory CLL, two for previously untreated AML, and one for MDS.

AbbVie and Roche are developing Venetoclax, the 38th breakthrough therapy designation for Roche’s portfolio of medicines and the 11th designation for its hematology portfolio.

In June 2020, Roche announced positive Phase 3 results from a study evaluating Venclexta in individuals with untreated AML who were ineligible for intensive induction chemotherapy.

The study, VIALE-A, found that Venclexta combined with azacitidine reduced the risk of death by 34 percent compared to azacitidine alone and led to higher composite complete remission rates, 66.4 percent compared to 28.3 percent.

The study also met its secondary endpoint of CR and CR with partial hematologic recovery, with the combination showing a CR + CRh of 64.7 percent compared to 22.8 percent with azacitidine alone

Based on positive trial results, FDA approved Venclexta in combination with azacitidine, decitabine, or low-dose cytarabine in October to treat newly diagnosed AML in adults 75 years or older.