Trying to nail down the actual number of new drugs approvals in 2013 is like trying to swat a fly — it can be highly elusive. Safe to say, however, as 2013 drew to a close, the total number of new did not match 2012’s high of 39.And yet, 2013 brought with it its own distinctions, including — launching new drugs at the fastest pace since the 1990s, according to the WSJ.
All-told, the FDA Center For Drug Evaluation (CDER) handed out approvals for a respectable 24 new molecular entities (NMEs) and biologics license application (BLAs) (not including any last-minute approval).
Other distinctions in 2013 were the approvals of two new breakthrough therapies (a new designation for the FDA to accelerate approval for drugs with hihgunmet medical needs); three NDAs with companion diagnostoics; and the approval of an antidody drug conjugate (ADC).
The honors for company front runners in the race for NMEs and BLA went to Bayer Healthcare, GlaxoSmithKline (GSK), Johnson & Johnson, Roche and Takeda, each with two or more new drug approvals
GSK: leader of the pack
GSK came in with three NME approvals—Breo Ellipta (fluticasone furoate and vilanterol trifenatate); Mekinist (trametinib dimethyl sulfoxide); and Tafinlar (dabrafenib mesylate).
Breo Ellipta is a combination of fluticasone furoate, an inhaled corticosteroid, and vilanterol, a long-acting beta, -adrenergic agonist, used to treat airflow obstruction and reducing exacerbations in patients with chronic obstructive pulmonary disease.
GSK also received FDA approval for two drugs to treat metastatic melanoma: Mekinist, a kinase inhibitor for treating unresectable or metaor V600K mutations as detected by an FDA-approved test, and Tafinlar, also a kinase inhibitor indicated for treating unresectable or metastatic melanoma with BRAF V600E mutation as detected by an FDA-approved test. In 2010, GSK formed a collaboration with the molecular diagnostics company bioMerieux to develop a companion diagnostic test to detect BRAF V600 (V600E and V600K) gene mutations found in several cancers, including melanoma. bioMerieux received FDA premarket approval of its companion diagnostic, THxID-BRAF, in May 2013.
Johnson & Johnson
By way of its bio/pharmaceutical units — Janssen Biotech and Janssen Pharmaceuticals — Johnson & Johnson was next across the finish line with two NMEs approvals in 2013.
Janssen Biotech partnered with the biopharmaceutical company Pharmacyclics for Imbruvica (ibrutinib), a kinase inhibitor for treating mantle-cell lymphoma (MCL), a rare type of blood cancer. Imbruvica was the second drug with breakthrough-therapy designation to receive FDA approval;
J&J’s second NME approval was for Invokana (canagliflozin), a sodium- glucose cotransporter 2 (SGLT2) inhibitor for treating Type 2 diabetes. Invokana is the first drug in a new class of SGLT2 inhibitors to be approved in the United States. Forxiga (dapagliflozin), developed by Bristol-Myers Squibb and AstraZeneca and also a SGLT2 inhibitor, was approved by the European Medicines Agency in November 2012. The companies submitted a new drug application for Foriziga to FDA in July 2013
While Imbruvica is the second drug with breakthrough-therapy designation to receive FDA approval, Roche’s Gazyva (obinutuzumab), a drug to treat leukemia (CLL), also approved in 2013, was the first.
The Food and Drug Administration Safety and Innovation Act, passed in July2012, gave FDA the ability to designate a drug as a breakthrough therapy at the request of the sponsor if preliminary clinical evidence indicated the drug may offer a substantial improvement over available therapies for patients with serious or life-threatening diseases.
In addition to Imbruvica, there are two other FDA-approved drugs to treat MCL: Velcade (bortezomib) by Millennium Pharmaceuticals, the oncology company of Takeda Pharmaceuticals, and Celgene’s Revlimid (lenalidomide).
Roche had two new BLAs approved by FDA so far in 2013: Gazyva and Kadxyla. Gazyva is a humanized anti-CD20 monoclonal antibody of the IgG1 subclass for treating CLL. It is produced by mammalian cell (Chinese hamster ovary Roche had two new BLAs approved by FDA thus far in 2013:
Gazyva (obinutuzumab) and Kadcyla (ado-trastuzumab emtansine). Gazyva is a humanized anti-CD20 monoclonal antibody (mAb) of the IgGI subclass for treating CLL. It is produced by mammalian cell (Chinese hamster ovary [CHO]) suspension culture.
Kadcyla is a HER2-targeted ADC, which contains the humanized anti-HER2 IgGl, trastuzumab, covalently linked to the microtubule inhibitory drug DM1 (a maytansine derivative) via the stable thioether linker MCC (4-[N-maleimidomethyl] cyclohexane-1-carboxylate). Emtansine refers to the MCC—DM1 complex. Trastuzumab is a well-characterized recombinant mAb produced by mammalian CHO cells, and the small-molecule components (DM1 and MCC) are produced by chemical synthesis. Kadcyla is the first Roche ADC approved by FDA.
The drug company scored with two NME approvals in 2013: Brintellix (vortioxetine hydrobromide) and Ne-sina (alogliptin benzoate). Brintellix, for treating major depressive disorder, was developed with H. Lundbeck. Nesina is a dipeptidyl peptidase-4 inhibitor designed to slow the inactivation of incretin hormones GLP-1 (glucagon-like peptide-1). GIP (glucose-dependent insulinotropic peptide) for treating Type 2 diabetes.
Two NMEs approved by FDA from Bayer were: Adempas (rio-ciguat) and Xofigo (radium RA-223 di-chloride). Adempas is a soluble guanylate cyclase stimulator to treat two forms of pulmonary hypertension. Xofigo is an alpha particle-emitting radioactive therapeutic agent for treating castration-resistant prostate cancer, symptomatic bone metastases, and no-known visceral metastatic disease. In 2009, Bayer partnered with Algeta for the development and commercialization of Xofigo.