The Class of 2012 for New Molecular Entities and Biologics

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Pharmaceutical Technology, Pharmaceutical Technology-12-02-2012, Volume 36, Issue 12

As 2012 comes to a close, Pfizer leads among Big Pharma companies for FDA approvals of new molecular entities and biologics.

O ne barometer of the health of the pharmaceutical/biopharmaceutical industry is new product development, and as 2012 comes to a close, how has the industry fared in this respect? In reviewing FDA approvals of new molecular entities (NMEs) and biologic license applications (BLAs) through Nov. 20, 2012, there are positive signs. FDA's Center for Drug Evaluation and Research (CDER) approved 31 NMEs and BLAs through Nov. 20, 2012, on par with the 30 NME and BLAs that it approved in 2011. The number of approvals, however, is not a sole determinant of success. A further look into the drug approvals for 2012 shows which companies may be graduating on the top of this year's class for new drug approvals.

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Leader of the pack

Of the 31 NMEs and BLAs approved by FDA's CDER thus far in 2012, 26 NMEs and 5 BLAs were approved (see Table I). Leading the large pharmaceutical companies was Pfizer, with four NME approvals. Pfizer received FDA approval for Bosulif (bosutinib monohydrate), a kinase inhibitor for treating adult patients with chronic, accelerated, or blast-phase Ph-positive chronic myelogenous leukemia. Pfizer also received approval for Elelyso (taliglucerase alfa), a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for adults with Type 1 Gaucher disease. Pfizer partnered with Protalix Biotherapeutics in developing Elelyso with the companies forming a collaboration in 2009. Pfizer gained approval for two more NMEs: Inlyta (axitinib), a kinase inhibitor for treating advanced renal cell carcinoma, and Xeljanz (tofacitinib), a Janus kinase inhibitor for treating adults with moderately to severely active rheumatoid arthritis with whom methotrexate did not work well (see Table I).

Patricia Van Arnum

Strong contenders


Six companies—Astellas, Eisai, Forest Laboratories, Roche/Genentech, Sanofi, and Teva—each had two new drug approvals thus far in 2012 (see Table I). Astellas had two NMEs approved in 2012: Myrbetriq (mirabegron) and Xtandi (enzalutamide). Myrbetriq is a beta-3 adrenergic agonist for treating overactive bladder. Xtandi, developed with Medivation, is an androgen receptor inhibitor for treating patients with metastatic castration-resistant prostate cancer who have previously received docetaxel.

Table I: FDA approvals of new molecular entities (NME) and biologic license applications (BLA) in 2012.*

Eisai's two NME approvals were for Belviq (lorcaserin hydrochloride) and Fycompa (perampanel). Belviq is a serotonin 2C receptor agonist indicated as an adjunct to a reduced-calorie diet and increased physical activity for chronic weight management. Belviq was developed by Arena Pharmaceuticals, a Zofingen, Switzerland-based company, for which it granted exclusive marketing and distribution rights to Eisai for most of North and South America. Fycompa is a noncompetitive AMPA glutamate receptor antagonist indicated as an adjunctive therapy for treating partial-onset seizures with or without secondarily generalized seizures in patients with epilepsy aged 12 years and older.

Forest Laboratories received approval for two NMEs in 2012: Linzess (linaclotide), developed with Ironwood Pharmaceuticals, and Tudorza Pressair (aclidinium bromide), developed with Almirall. Linzess is a guanylate cyclase-C agonist for treating irritable bowel syndrome or chronic idiopathic constipation. Tudorza Pressair is an anticholinergic indicated as a long-term maintenance treatment of bronchospasm associated with chronic obstructive pulmonary disease.

Roche/Genentech also had two new drug approvals, one for a NME, Erivedge (vismodegib), and one for a BLA, Perjeta (pertuzumab). Erivedge is a hedgehog pathway inhibitor for treating metastatic basal-cell carcinoma or locally advanced basal-cell carcinoma that has recurred following surgery or for pateints who are not candidates for surgery or radiation. Perjeta is a HER2/neu receptor antagonist indicated in combination with trastuzumab and docetaxel for treating patients with HER2-positive metastatic breast cancer.

Sanofi received approval for its NME Aubagio (teriflunomide) and its BLA Zaltrap (ziv-aflibercept). Aubagio is a pyrimidine synthesis inhibitor for treating patients with relapsing forms of multiple sclerosis. Zaltrap is a recombinant fusion protein, which acts as a soluble receptor that binds to vascular endothelial growth factor-A (VEGF-A), VEGF-B, and placental growth factor and is used to treat metastatic colorectal cancer. Sanofi partnered with the biopharmaceutical company Regeneron Pharmaceuticals for Zaltrap.

The generic-drug company Teva had two approvals, one for its NME Synribo (omacetaxine mepesuccinate), a drug to treat leukemia, and a BLA for tbo-filgrastim, which was approved as an original BLA and not as a biosimiliar to Amgen's Neupogen (filgrastim), which is a previously approved biologic. Tbo-filgrastim is a human granulocyte colony-stimulating factor produced by recombinant DNA technology for reducing neutropenia in patients with non-myeloid malignancies.

Other companies

Approvals constitute a mixed bag for other large companies. Merck & Co. gained one new drug approval in 2012 for Zioptan (tafluprost ophthalmic solution), a prostaglandin analog indicated for reducing elevated intraocular pressure in patients with open-angle glaucoma or ocular hypertension. Bayer received FDA approval for Stivarga (regorafenib), a kinase inhibitor for treating metastatic colorectal cancer. Eli Lilly's subsidiary Avid Radiopharmaceuticals received approval for the radioactive diagnostic agent, Amyvid (florbetapir F-18). Takeda Pharmaceutical and Affymax received approval for Omontys (peginesatide acetate), an erythropoiesis-stimulating agent to treat anemia.

Equally important in evaluating R&D productivity is seeing which companies did not receive FDA approval for NMEs or new biologics (excluding vaccines). Through Nov. 20, 2012, GlaxoSmithKline, Novartis, AstraZeneca, Bristol-Myers Squibb, and Abbott were among the large companies without a NME or original BLA FDA approval this year. As the industry waits for 2012 to come to a close, it has yet to be seen if their late-stage candidates under review will get the FDA nod this year.

Patricia Van Arnum is a executive editor of Pharmaceutical Technology, 485 Route One South, Bldg F, First Floor, Iselin, NJ 08830 tel. 732.346.3072, twitter@PharmTechVArnum