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FDA Drug Approvals

2013 FDA drug approvals

Posted on March 3, 2014 by pbeswick

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This analysis by Asher Mullard published in Nature Reviews Drug Discovery (2014,13, 85-89) reports the new drugs approved by FDA in 2013. From a total of thirty-six applications, twenty-five new small molecules and two new biologics were approved. The same trend as the previous years was overall maintained, with the exception of 2012. (A notable achievement was the high approvals (33%) of new molecular entities for the treatment of orphan disease. In addition, 33% of the new approvals had a unique mode of action and were identified as first-in-class agents. The anticancer therapeutic area obtained the majority of approvals (eight, six of which are for orphan indication), followed by metabolic and endocrinology, antiviral and medical imaging (three approvals for each category). Cardiology, neurology, respiratory and women’s health have two agents approved each, and only one new approval for psychiatry and dermatology.

Ten drugs received a priority review status, which is given to drugs that potentially have significant advances in term of safety or effectiveness for the treatment, diagnosis or prevention of serious conditions, including: ado-trastuzumab emtansine, radium RA 223 dichloride, afatinib, obinutuzumab, and ibrutinib (anticancer); dolutegravir, simeprevir, and sofosbuvir (antiviral); gadoterate meglumine (diagnostic); and riociguat (cardiology). Among these, the humanized CD-20-specific monoclonal antibody obinutuzumab (chronic lymphocytic leukaemia), the Bruton’s tyrosine kinase inhibitor ibrutinib (mantle cell limphona) and the HCV nucleotide analogue NS5B polymerase inhibitor sofosbuvir (chronic HCV infection as a part of an antiviral treatment regimen), received also the breakthrough designation status. This status is given to drugs used to treat serious or life-threatening disease or condition and that showed a substantial improvement versus existing therapies as evidenced on early clinical data.

Thirteen new potential blockbusters have been forecast by 2018-2019 (Thomson Reuters Cortellis) (Figure 2) including:

- Sofosbuvir (Gilead), first-in-class HCV nucleotide analogue polymerase inhibitor, with an annual potential of 6.8 billions $US;
- Dimethyl fumarate (Biogen Idec), approved for the treatment of multiple sclerosis with an unknown mechanism of action, with sale forecasts of 6 billions $US;
- Ibrutinib (Pharmacyclics), first-in-class Bruton’s tyrosine kinase inhibitor, approved for the treatment of mantle cell limphoma, with an annual potential of 4.5 billions $US;
- Ado-trastuzumab emtansine (Genentech), an antibody-drug conjugate approved for the treatment of HER2-positive metastatic breast cancer, with a potential income of 4.1 billions $US;
- “Umeclidinium and vilanterol” and “Fluticasone and vilanterol” (GSK) both approved for the treatment of chronic obstructive pulmonary disease, with sale forecasts of 3.1 and 2.8 billions $US respectively.

In terms of companies, GSK had the highest number of approvals (five, including the HIV-integrase inhibitor dolutegravir develop with ViiV) within three different therapeutic areas, and with three potential blockbusters.
Despite a 31% drop of approvals compared to the previous year, 2013 saw the approvals of innovative new products that will have a significant impact for medical care. Analysts forecast thirteen new potential blockbusters (almost half of all approvals), and six of them may have a multibillion dollar potential