Bimagrumab 3rd Novartis drug to win FDA 'breakthrough' designation
This article was originally published in Scrip
Novartis has won its third breakthrough therapy designation from the US FDA, which granted the status to the Swiss drug maker's investigational muscle-wasting disease drug bimagrumab (BYM338), a fully human monoclonal antibody.
Shares of Novartis jumped 3%, or $2.23, on the New York Stock Exchange in morning trading on 20 August.
The breakthrough designation is intended to help expedite the US regulatory process – providing companies the opportunity to meet with the agency's review team throughout the development of a drug and receive timely advice and interactive communication to ensure the medicine's investigational program is as efficient as practicable (scripintelligence.com, 26 June 2013).
To obtain the breakthrough therapy designation, preliminary clinical evidence must indicate the experimental drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical request. In other words, companies must be able to show with data they've earned the breakthrough therapy status.
Novartis earlier had gained the breakthrough status for its experimental non-small-cell lung cancer (NSCLC) drug LDK378, a highly selective anaplastic lymphoma kinase (ALK) inhibitor, and its investigational acute heart failure drug serelaxin (RLX030) (scripintelligence.com, 15 March 2013, 22 June 2013).
For bimagrumab, Novartis said the breakthrough designation highlights the drug's potential to address an unmet medical need in a serious disease – sporadic inclusion body myositis (sIBM), which can be life-threatening.
Although it is rare, sIBM is the most common degenerative disease of muscle in adults older than 65 years, Novartis pointed out.
The condition is characterized by a slowly progressive, asymmetric, atrophy and weakness of muscles. Patients with sIBM usually become wheelchair bound within 10 to15 years of disease onset.
Patients with sIBM often gradually lose the ability to walk, resulting in falls and injuries. They also often lose hand function and have swallowing difficulties.
If approved, bimagrumab, which was developed to treat pathological muscle loss and weakness, has the potential to be the first treatment for sIBM patients, Novartis noted.
Bimagrumab works by binding with high affinity to type 2 activin receptors, preventing natural ligands from binding, including myostatin and activin.
The drug, which is administered by intravenous infusion, stimulates muscle growth by blocking signaling from these inhibitory molecules, the Basel, Switzerland-based pharma said.
The breakthrough designation for bimagrumab, which was developed by the Novartis Institutes for Biomedical Research in collaboration with Morphosys, whose HuCAL library was used to identify the antibody, was based on the results of a Phase II proof-of-concept study, which showed the experimental medicine substantially benefited patients with sIBM over placebo.
Novartis said it plans to present the results of the Phase II study on 14 October at the annual meeting of the American Neurological Association, with the data expected to be published in a major medical journal later this year.
In addition to being developed for sIBM, bimagrumab is in clinical development for chronic obstructive pulmonary disease, cancer cachexia, sarcopenia and in mechanically ventilated patients.
As the third of Novartis' drugs to receive the breakthrough designation, bimagrumab stands as an example of the company's "commitment to innovation addressing significant unmet medical needs and enhancing the lives of patients," said Dr Timothy Wright, global head of development at the firm.
According to the FDA's most recent figures, the Center for Drug Evaluation and Research has granted breakthrough designation to 25 of the 77 requests it has received, denying 27. The Center for Biologics and Research has not yet granted any of the eight requests it has received, denying seven of them so far, with one still pending.
Earlier this month, Ariad revealed its tyrosine kinase inhibitor AP26113, which is under investigation to treat ALK+NSCLC, was one of the medicines snubbed by the FDA (scripintelligence.com, 09 August 2013).
In addition to Novartis, other firms that have won the breakthrough therapy designation include AbbVie, Alexion, Bristol-Myers Squibb, Genentech, Genmab, GlaxoSmithKline, Janssen, Merck, Pfizer, Pharmacylics, Synageva and Vertex (scripintelligence.com, 7 May 2013, 29 May 2013, 26 April 2013, 16 May 2013, 2 May 2013, 24 April 2013, 27 June 2013, , 11 April 2013, 9 April 2013, 14 February 2013, 20 May 2013, 8 January 2013).