Amgen Leukaemia Drug Zooms to US Launch

December 04, 2014 Amgen leukaemia drug zooms to US launch

Jonathan Gardner

Amgen’s new cancer drug Blincyto will not be a record-breaker in generating sales, but it may be remembered for another accomplishment: one of the fastest approvals on record.

US regulators took just over two months to pass the leukaemia agent’s papers across the necessary desks to achieve marketing authorisation yesterday, more than five months ahead of the agency’s decision deadline. Rapid action is a sign of how winning breakthrough designation benefits drugmakers; an analysis of quickest approvals also illustrates the draw of developing cancer drugs (see table below).

Record breakers

Blincyto was approved in relapsed or refractory Philadelphia chromosome-negative precursor B-cell acute lymphoblastic leukaemia, a condition that affects about 6,000 US patients a year. It is not forecast to be a huge seller – EvaluatePharma’s consensus forecast puts sales of $181m in 2020 – and thus the antibody can hardly be called a game-changing agent on the commercial side.

However, a lack of regulatory complication following a high-speed clinical programme explains the attraction of the agent known generically as blinatumomab. Amgen acquired Blincyto with the $1.1bn buyout of Micromet nearly three years ago, and the California-based group has achieved approval on data from 185 patients – a 400-patient confirmatory study with an overall survival endpoint is now underway.

Treatment protocols for resistant disease now involve chemotherapy and bone marrow transplants, so new treatment options are welcome. As the following post-2007 analysis of fast approvals shows, one of the best ways to get the agency to wave a drug through is to prove efficacy in haematological cancers. The FDA's fastest work since 2007

Approval FDA Indication (pharma Review Product Company Time Approval class) Status (Mths) Date

Breakthrough Blincyto ALL (anti-CD19 MAb) Amgen 2.3 03/12/2014 therapy

Priority Jevtana Prostate cancer (taxane) Sanofi 2.6 17/06/2010 review

ALL/CLL (BCR-ABL & pan- Priority Iclusig ARIAD Pharmaceuticals 2.6 14/12/2012 FGFR inhibitor) review

Prostate cancer (androgen Astellas Priority Xtandi 3.3 31/08/2012 receptor antagonist) Pharma/Medivation review

Cystic fibrosis ((CFTR Breakthrough Kalydeco Vertex Pharmaceuticals 3.5 31/01/2012 potentiator) therapy

Melanoma (B-Raf kinase Priority Zelboraf Roche/Daiichi Sankyo 3.6 17/08/2011 inhibitor) review

Anti-thrombin III deficiency Priority ATryn Ovation Pharmaceuticals 4.0 06/02/2009 (blood product) review

Breakthrough Zykadia Lung cancer (ALK inhibitor) Novartis 4.1 29/04/2014 therapy

Prostate cancer (CYP17 Priority Zytiga Johnson & Johnson 4.2 28/04/2011 inhibitor) review

Mantle cell lymphoma Johnson Priority Imbruvica 4.5 13/11/2013 (BTK inhibitor) & Johnson/Pharmacyclics review

Ariad Pharmaceuticals’ Iclusig and Pharmacyclics’ and Johnson & Johnson’s Imbruvica also feature on this list. Gleevec’s 2001 approval in chronic myeloid leukaemia was also speedy, requiring just 2.4 months of review before the agency said yes to Novartis (FDA approval times continue to shorten, March 11, 2011).

Cancer drugs dominate the list, taking eight of the 10 quickest approvals. To whip through the FDA at an oncological speed a candidate must offer a very significant improvement in treatment for an underserved disease – see Vertex Pharmaceuticals’ cystic fibrosis drug Kalydeco.

Worth it?

Quick path to market aside, Amgen will need Blincyto to outperform expectations to justify the billion-dollar price it paid for Micromet – or at least Micromet’s bispecific T-cell engager (BiTE) technology offers other strings for Amgen’s bow.

Current forecasts give Blincyto a net present value of $699m. What is more, it is playing in the same space as the chimaeric antigen receptor therapy (CART) projects now in development, and thus Amgen needs to hope its data match up well with Juno Therapeutics’ CD-19 targeting agent, for example. No doubt Blincyto’s full phase II data will be examined thoroughly (ASH preview – Stock-moving for some, damp squib for others, November 28, 2014).

Blincyto will be remembered for its breakneck approval, a sign that it is a marked improvement on existing therapy. It would be a huge disappointment if Amgen cannot turn the scientific edge into a commercial one.

To contact the writer of this story email Jonathan Gardner in London at [email protected] or follow @JonEPVantage on Twitter