Honor Roll for the Class of 2015
Our annual year-end wrap-up of new drug approvals and launches indicates that 49 new molecular entities (NMEs) and biologic drugs had been introduced in their first markets worldwide as of December 15, 2015. Another 29 significant line extensions, including new indications, new formulations or new combinations of previously marketed products, also reached their first markets this year. Twenty-one NMEs and line extensions had been approved but not yet launched as of mid-December. Although these numbers are indicative of the good overall health of the pharma and biotech industry, there was a notable drop in new launches with respect to the previous year, which saw 58 new product intros. Nonetheless, quality is more important than quantity when it comes to new drugs, and the class of 2015 has some truly outstanding members.
HIGHEST NUMBER OF NEW CANCER DRUGS IN A DECADE
This year saw the approval and launch of four new drugs for multiple myeloma, a hematologic malignancy that until a decade ago had only a handful of treatment options. In the words of Walter Capone, president of the Multiple Myeloma Research Foundation, 2015 was a ‘watershed year’ for the 90,000 U.S. patients with the rare blood cancer.
Although multiple myeloma is the clearest example, cancer patients as a group had plenty to cheer about this year, which recorded the highest number of new cancer drugs in a decade. All told, 14 new cancer drugs and biologics were launched for the first time worldwide.
NEW DRUGS FOR PREVIOUSLY UNTREATABLE DISEASES
Several drugs were launched this year for previously untreatable disorders, some quite common and others extremely rare. One such drug is Shire’s Vyvanse® (lisdexamfetamine dimesylate), the first treatment for moderate to severe binge eating disorder (BED) in adults. An estimated 2.8 million U.S. adults struggle with BED, the most common eating disorder in that country.
U.S. patients are also the first to benefit from Addyi™ (flibanserin), the first treatment for hypoactive sexual desire disorder in women. The FDA finally approved the product this year, after having rejected the NDA on two previous occasions, albeit with strict limitations on use given its potentially serious side effects. The approval proved controversial, and the FDA recently defended its decision in the New England Journal of Medicine.
For those with hereditary factor X disorder, a rare bleeding disorder that affects just 400-600 patients in the U.S., 2015 will be remembered as the year in which a specific drug therapy finally became available: BPL’s Coagadex® (coagulation factor X [human]). The year also saw the approval and launch in several countries of Alexion’s asfotase alfa (Strensiq®), the first treatment for the life-threatening, ultra-rare metabolic disorder hypophosphatasia.
In another historic first, Sanofi Pasteur’s tetravalent dengue vaccine was approved for the first time in Mexico in mid December. Two decades after Sanofi initiated the search for an effective dengue vaccine, Mexican authorities gave the OK for Dengvaxia®, indicated for the prevention of disease caused by all four dengue virus serotypes in preadolescents, adolescents and adults living in endemic areas. Dengue is endemic in 125 countries and sickens an estimated 50-100 million worldwide each year. Denvaxia will be launched in 2016.
A NEW CROP OF FIRST-IN-CLASS AGENTS
Eleven novel products with first-in-class mechanisms of action were launched this year, including two novel agents for hypercholesterolemia. Approximately a decade ago, inhibition of the circulating protein proprotein convertase subtilisin/kexin type 9 (PCSK9) was identified as a promising new strategy for lower LDL cholesterol in patients who do not respond to statins, the standard therapy. PCSK9 exerts its hypercholesterolemic effect by promoting degradation of the LDL receptor. Its absence leads to lower LDL cholesterol and reduced cardiovascular risk. The race to develop a first-in-class PCSK9 inhibitor culminated this summer in the nearly simultaneous approval of two anti-PCSK9 monoclonal antibodies (MAbs): evolocumab (Repatha™; Amgen) and alirocumab (Praluent®; Regeneron/Sanofi).
Other first-in-class agents launched from the class of 2015 include the anti-IL-5 MAb mepolizumab (Nucala®; GlaxoSmithKline) for asthma; the anti-IL-17A MAb secukinumab (Cosentyx™; Novartis), launched for psoriasis and psoriatic arthritis and approved for ankylosing spondylitis; the anti-SLAMF7 MAb elotuzumab (Empliciti™; AbbVie/Bristol-Myers Squibb) for multiple myeloma; and the cyclin-dependent kinase (CDK) 4/6 inhibitor palbociclib (Ibrance®; Pfizer) for breast cancer. These and more products and trends will be covered in depth in “The Years New Drugs & Biologics”, an annual feature of the Thomson Reuters journal Drugs of Today, currently in preparation.