Amgen leukaemia drug zooms to US launch

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
Product Indication (pharma class) Company Review Status Approval Time (Mths) FDA Approval Date
Blincyto ALL (anti-CD19 MAb) Amgen Breakthrough therapy 2.3 03/12/2014
Jevtana Prostate cancer (taxane) Sanofi Priority review 2.6 17/06/2010
Iclusig ALL/CLL (BCR-ABL & pan-FGFR inhibitor) ARIAD Pharmaceuticals Priority review 2.6 14/12/2012
Xtandi Prostate cancer (androgen receptor antagonist) Astellas Pharma/Medivation Priority review 3.3 31/08/2012
Kalydeco Cystic fibrosis ((CFTR potentiator) Vertex Pharmaceuticals Breakthrough therapy 3.5 31/01/2012
Zelboraf Melanoma (B-Raf kinase inhibitor) Roche/Daiichi Sankyo Priority review 3.6 17/08/2011
ATryn Anti-thrombin III deficiency (blood product) Ovation Pharmaceuticals Priority review 4.0 06/02/2009
Zykadia Lung cancer (ALK inhibitor) Novartis Breakthrough therapy 4.1 29/04/2014
Zytiga Prostate cancer (CYP17 inhibitor) Johnson & Johnson Priority review 4.2 28/04/2011
Imbruvica Mantle cell lymphoma (BTK inhibitor) Johnson & Johnson/Pharmacyclics Priority review 4.5 13/11/2013

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

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