Another busy weekend for Celgene saw the firm get European approval for Revlimid as a treatment for a rare form of blood disease and present more promising data on its multiple myeloma drug pomalidomide.
First up, the European Commission has expanded approval on Revlimid (lenalidomide) to allow treatment of patients with transfusion-dependent anaemia due to low or intermediate-1 risk myelodysplastic syndromes (MDS) with an isolated chromosomal abnormality called deletion 5q when other options do not work. MDS is a type of cancer where the production of blood cells and platelets by the bone marrow is disrupted, which can often lead to severe anemia, infections and bleeding.
Some 50% of individuals with MDS will have some form of cytogenetic, or chromosome, abnormality, and 30% of those are likely to have the specific del (5q) abnormality. It is associated with a poor prognosis including the risk of progressing to acute myeloid leukaemia, which is often fatal.
Ari Giagounidis of the Marien Hospital in Dusseldorf, said that the standard of care for MDS has historically been red blood cell transfusions, "which can help control the disease but pose a tremendous burden to patients – particularly the elderly, who make up the majority of MDS patients – as well as to healthcare providers and health systems". Speaking to PharmaTimes World News on the fringes of the European Hematology Association meeting in Stockholm, he said that taking the once-a-day pill will "transform the landscape" of treatment for these MDS patients.
Dr Giagounidis went on to say that a trial for non-deletion 5q and Revlimid is fully enrolled, while the drug is expected to be launched in Germany first. Approval has already been granted in the USA for both low and high-risk MDS patients. Revlimid, Celgene's biggest earner, is best-known as a treatment, in combination with dexamethasone, for multiple myeloma.
Indeed, at EHA, updated results were presented on Celgene's follow-up to Revlimid, pomalidomide, from the MM-003 study that compares the drug in combination with low-dose dexamethasone with high dose dexamethasone in MM patients with late-stage disease who have previously failed on Revlimid and Takeda and Johnson & Johnson's Velcade (bortezomib). Both progression free survival (PFS) and overall survival (OS) significantly improved with pomalidomide and low-dex, compared to high-dose dex (PFS: 4.0 months vs 1.9 months; OS: 12.7 vs 8.1 months).
Pomalidomide is already approved in the USA as Pomalyst and a green light is expected on this side of the Atlantic shortly, given that the European Medicines Agency's Committee for Human Medicinal Products issued a positive opinion a month ago. Xavier Leleu of the Hopital Claude Huriez in Lille, France told PharmaTimes World News noted that pomalidomide is consistently showing 30% response rates and "we are getting green signals all over the board".
Noting that patients are old and fragile, he noted that given its safety and efficacy shows that since Revlimid and Velcade, pomalidomide "is simply better than any other drug we have had".