Pharma Strategy Blog

Last night and this morning brought some topical news on the leukemia front.

Just before 5pm EST yesterday, the FDA announced they had approved Roche/Genentech's rituximab (Rituxan) in chronic lymphocytic leukemia (CLL).  Rituxan is already approved for non-Hodgkins Lymphoma (NHL) and rapidly the standard of care when combined with CHOP chemotherapy in this disease.  CLL is the most common adult leukemia and tends to affect older patients.

According to the Roche press release this morning, the FDA approval was for:

"Rituximab plus fludarabine and cyclophosphamide (FC) chemotherapy for people with either previously untreated (first-line) or previously treated (relapsed or refractory) CD20-positive chronic lymphocytic leukemia (CLL)."

The CLL data was presented last year at ASH, showing that FCR led to improved survival compared to FC alone.  FDA's press release highlighted the results:

"The safety and effectiveness of Rituxan was evaluated in two studies that measured progression-free survival, defined as the time a patient in the study lived without the cancer progressing.

In one study of 817 patients who had not received any prior chemotherapy, progression-free survival was eight months longer for those receiving Rituxan plus chemotherapy than for those who received chemotherapy alone.  In another study of 522 persons whose cancer had progressed following other chemotherapy drugs, progression-free survival was five months longer for those who received Rituxan plus chemotherapy.

The FDA analyzed the data on patients 70 years of age and older who had received Rituxan and found no evidence that adding the drug to chemotherapy benefitted elderly patients compared to receiving chemotherapy alone.  However, there was also no evidence that Rituxan was harmful to elderly patients."

This approval affects several companies in the CLL space. 

Late last year, GSK received approval for their CD20 antibody, ofatumumab (Arzerra) in CLL in the refractory setting but are likely to be impacted by rituximab's approval in both first and second line CLL.  My guess would be that physicians will be very comfortable and experienced using the Roche/Genentech monoclonal antibody and therefore using it in CLL as well as NHL will not be a difficult stretch. 

Bendamustine (Treanda), from Cephalon, is an old drug that has been making a comeback in NHL and after the success of the bendamustine-rituximab (BR) combination in NHL that was presented at ASH, it may well become the new standard of care there instead of R-CHOP.  Trials are ongoing with BR in CLL and if positive, I can see that becoming a solid option in CLL in the not too distant future.  For now, FCR looks like being the most efficacious first-line option in CLL, replacing FC.  It may not be long before BR offers similar or better efficacy than FCR with fewer side effects.  We will have to wait and see.

The other news that was interesting in my inbox is that Novartis (a client), announced that nilotinib (Tasigna) received FDA priority review for newly diagnosed patients with early-stage chronic myeloid leukemia (CML):

"FDA priority review status is granted to therapies that offer major advances in treatment or provide a treatment where no adequate therapy exists.  This status accelerates the standard review time from 10 to six months.  Tasigna demonstrated that significantly fewer patients progressed to more advanced stages of the disease than the standard of care Glivec® (imatinib)* at 12 months.  Tasigna also showed a statistically significant improvement over Glivec in every other measure of efficacy in the trial, including major molecular response (MMR) and complete cytogenetic response (CCyR) at 12 months."

Novartis and BMS were in a race to file for the front-line indication for their second generation tyrosine kinase inhibitors (TKIs) with comparative trials versus the standard of care, imatinib (Gleevec/Glivec).  Novartis presented the initial Tasigna results at ASH in December in a late breaking abstract that pointed to earlier and deeper responses with nilotinib compared with imatinib.

BMS didn't presented any data from their randomised registration trial at the meeting, but recently announced at the JP Morgan Healthcare conference last month, that they expected data to be available at ASCO.  I'm not sure whether they submitted a regular abstract or a late breaker, but it will be interesting to see what the data looks like and the big question now is when will they be filing Sprycel with the FDA for the front-line indication?

It going to be an interesting few months ahead in leukemia, that's for sure!