93 New Medicines 2013

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FDA regulators during 2012 approved 39 new molecular entities, the highest amount in one year since 1997.

U.S. regulators last year approved for marketing 93 new medicines according to Med Ad News criteria. This figure represents an additional 13 products versus the 2011 amount of 80. Previous years’ totals of new drug approvals by the Food and Drug Administration numbered 88 in 2010, 92 during 2009, 85 for 2008, and 73 in 2007.

The total of approved new molecular entities filed under new drug applications and therapeutic biologics submitted through original biologic license applications rose to 39 during 2012 compared to 30 in 2011. The Food and Drug Administration’s Center for Drug Evaluation and Research gave the green light to 21 NMEs/new BLAs in 2010, 26 for 2009, 24 during 2008, and 18 in 2007. The 2012 amount was the highest since 39 NMEs/BLAs were approved in 1997, which followed a one-year record of 53 during 1996.

The two-year increase in new molecule approvals is significant during which time the industry has experienced the patent-expiration losses of some of the best-selling prescription medicines ever. The industry’s total branded drug sales are undergoing an annual decline as generic competition continues to increase, development costs rise, and jobs decrease.

The 39 NME/BLA approvals of 2012 include about a dozen oncology-related products. That grouping includes Perjeta, a personalized medicine designed to specifically block the HER2 protein on the surface of some cancer cells. This Roche/Genentech biologic antibody is believed to work by attaching to HER2 receptors to stop signals that make tumor cells grow and divide, and additionally by signaling the body’s immune system to destroy cancer cells. The drug is approved in combination with the Roche/Genentech mega-blockbuster Herceptin (trastuzumab) and docetaxel chemotherapy for treating individuals with HER2-positive metastatic breast cancer who have not received previous anti-HER2 therapy or chemotherapy for metastatic disease. Industry trackers have forecasted Perjeta global sales of nearly $2.5 billion for 2018.

Another anticipated annual billion-dollar sales generator from the Class of 2012 New Medicines is Xeljanz. Composed of the active ingredient tofacitinib, the new drug was FDA-approved for treating adults with moderately to severely active rheumatoid arthritis (RA) who have had an inadequate response or intolerance to methotrexate. The Pfizer product represents the first approved RA treatment in a new class of medicines called Janus kinase (JAK) inhibitors and the first new oral disease-modifying antirheumatic drugs for the disease in more than a decade. Xeljanz is available as a second-line medicine for rheumatoid arthritis, thus treatment with a biologic is not necessary before taking it. Various analyst groups have projected more than $1-plus billion in global Xeljanz sales by 2016.

Approved by FDA in August 2012, the HIV medicine Stribild has been predicted to approach $3 billion in 2018 global sales. Marketed by Gilead Sciences, the complete once-daily single tablet regimen is intended for HIV-1 infection for treatment-naïve adults. Stribild unites four compounds in one daily tablet: the integrase inhibitor elvitegravir, the pharmacoenhancing agent cobicistat, and the nucleoside analog reverse transcriptase inhibitors emtricitabine and tenofovir disoproxil fumarate.

Perhaps the medicine with the highest sales potential to be cleared for marketing during 2012 is Eliquis. Approved by the U.S. regulatory agency at the end of 2012, Eliquis is intended to reduce the risk of stroke and systemic embolism in patients with nonvalvular atrial fibrillation. Containing the active chemical apixaban, the oral Factor Xa inhibitor anticoagulant is jointly marketed by Pfizer and Bristol-Myers Squibb. By inhibiting the key blood clotting protein Factor Xa, Eliquis decreases thrombin generation as well as blood clot formation. Some industry insiders have projected more than $4.7 billion in 2018 global sales for the drug. For more details about Eliquis, please see the Med Ad News Best New Medicine story on page ??.

In addition to Xeljanz and Eliquis, New York-based Pfizer is the marketer behind several other new molecular entities approved during 2012. Bosulif (bosutinib) was approved for treating adult patients with chronic, accelerated, or blast phase Philadelphia chromosome-positive chronic myelogenous leukemia (CML) with resistance, or intolerance to prior therapy. This kinase inhibitor limits cancer cell growth by inhibiting the Abl and Src signaling pathways. The once-daily medicine represents the only therapy FDA-approved with pivotal clinical-study data that included CML patients treated with imatinib followed by a second-generation tyrosine kinase inhibitor.

Another Pfizer orphan drug cleared for approval by U.S. regulators during 2012 was Elelyso (taliglucerase alfa). The product is available for long-term enzyme replacement therapy to treat a form of Gaucher disease, a rare genetic disorder. Elelyso injection replaces the missing enzyme in patients with a confirmed diagnosis of Type 1 (non-neuropathic) Gaucher disease. This represents the first FDA-approved plant cell-based enzyme replacement therapy for Gaucher disease. Elelyso additionally is the first approved plant cell-expressed drug derived from ProCellEx, a proprietary manufacturing system from Protalix BioTherapeutics that uses genetically engineered carrot cells.

The kinase inhibitor Inlyta was approved by FDA during January 2012 patients with a type of advanced kidney cancer. The medicine is available for treating advanced renal cell carcinoma (RCC) after failure of one prior systemic therapy. The Pfizer oral therapy is designed to inhibit tyrosine kinases, including vascular endothelial growth factor (VEGF) receptors 1, 2 and 3. Those three receptors can influence tumor growth, vascular angiogenesis, as well as progression of cancer (the spread of tumors).

Pfizer launched Quillivant XR in the United States during January 2013 for treating attention deficit hyperactivity disorder. This is the first once-daily, extended-release liquid methylphenidate for ADHD. Quillivant XR gained U.S. regulatory clearance in September 2012 for ADHD patients aged 6 years and older. The product was developed in alliance with NextWave Pharmaceuticals’ manufacturing partner Tris Pharma using the latter’s patent-protected drug-delivery platform. NextWave was acquired by Pfizer on Nov. 27, 2012.

An oral suspension formulation of Revatio was FDA-approved on Aug. 30, 2012. The new formulation is indicated for treating pulmonary arterial hypertension (WHO Group 1) in adults to improve exercise ability and delay clinical worsening. Revatio was first approved by U.S. regulators in tablet form during June 2005, and the injectable version was given marketing clearance in November 2009.

Stendra’s FDA approval during April 2012 represented the first new prescription medicine approved in nearly a decade for erectile dysfunction. Containing the main ingredient avanafil, the phosphodiesterase type 5 inhibitor is available as a tablet for the roughly 30 million American men suffering from ED. Licensed from Mitsubishi Tanabe Pharma, Vivus holds global (except certain Asian Pacific Rim countries) development and commercial rights to Stendra for treating sexual dysfunction. The product is marketed in South Korea by JW Pharma under the trade name Zepeed.

Myrbetriq is the first oral overactive bladder treatment with a distinct mechanism of action since the market introduction of anticholinergic agents in roughly 30 years. Composed of (mirabegron), the extended-release tablet drug was approved in April 2012 for treating overactive bladder with symptoms of urge urinary incontinence, urgency and urinary frequency. The once-per-day beta-3 adrenergic agonist was discovered and developed by Astellas Pharma. Myrbetriq was studied extensively in more than 10,000 individuals over a decade. The product offers a new treatment option for OAB patients as antimuscarinics serve as the current treatment standard. Antimuscarinics function by binding to muscarinic receptors in the bladder and inhibiting involuntary bladder contractions. Myrbetriq relaxes the detrusor smooth muscle during the storage phase of the urinary bladder fill-void cycle by activation of beta-3 adrenergic receptors that improves bladder capacity.

Another April 2012 approval by FDA was Amyvid, the first radioactive diagnostic agent cleared for PET imaging of beta-amyloid neuritic plaques in the living brain. Amyvid is indicated for brain imaging of beta-amyloid plaques in patients with cognitive impairment who are being evaluated for Alzheimer’s disease and other cognitive decline causes. Amyvid binds to amyloid plaques, a hallmark characteristic of AD, and is detected via PET scan images of the brain. Eli Lilly and its wholly owned subsidiary Avid Radiopharmaceuticals announced the product’s U.S. launch on June 1, 2012.

Notes And Methodology

This annual special report features the new prescription medicines approved in the United States during 2012. The information was gathered from pharmaceutical/biotechnology companies, the Food and Drug Administration, and the fi les of Med Ad News.The 93 new medicines detailed in this special report were approved by FDA regulators through a new drug application or biologics license application. The new medicines may include new molecular entities, biotechnology drugs, biologicals, imaging agents, branded generic drugs, and branded new formulations of existing products. Exclusions from this list include medical devices, nonbranded generic drugs approved via an abbreviated new drug application, over-the-counter drugs, tentative approvals, and new indications for alreadymarketed drugs. All medicines were approved for U.S. marketing from Jan. 1, 2012, through Dec. 31, 2012.