The recent FDA approval of Ibsrela (tenapanor), Ardelyx Inc.’s treatment for irritable bowel syndrome with constipation in adults, brings, according to the agency’s data, the number of new molecular entities (NMEs) to 27 this year. With just three months remaining, it appears that the biopharma sector is on pace with the five-year average of about 43 NMEs approved annually. Interestingly, less than half (12) of those 2019 new medicines have been brought to market by big pharma companies. It appears that innovative success, in terms of new medicines approved, continues to be skewed in favor of biotech companies. Last year, for example, biotechs gained 39 of the record 59 FDA NME approvals.
Leading biotech company Gilead Sciences Inc. is representative of that evolving success, and its leadership in the antiviral space along with the approval of new blockbuster products helped the Foster City, Calif.-based company top the annual Pharmaceutical Innovation Index (PII) for the first time. Released by Idea Pharma earlier this year, the index, now in its ninth year, aims to provide an objective assessment of how well the top 30 biopharma companies perform in their ability to bring products from early phase to market, and to commercialize them successfully. (See Pharmaceutical Innovation Index Top 10, right.)
Combination of factors
According to Idea Pharma, a combination of factors helped push Gilead into the top position this year, including an outstanding freshness index score, that measures how much of a company’s sales come from products approved within the past five years. Gilead’s 60% rating was more than twice its nearest rivals and compares to an average across the top 30 biopharma companies of 11%.
The company was cited as being committed to its new blockbuster HIV treatment, Biktarvy (bictegravir 50 mg/emtricitabine 200 mg/tenofovir alafenamide 25 mg).
In its second-quarter financial report, the company recorded sales of $4 billion from its HIV products, compared to $3.7 billion for the same period last year. The increase was primarily driven by higher sales volume as a result of the continued uptake of Biktarvy.
Among the rankings, Eli Lilly and Co. was the leading gainer, jumping 10 positions to take the third spot. Sanofi SA gained nine places. Sanofi, in fact, has taken advantage of acquiring strategic biotech assets to boost its pipeline. A most recent example of that came in February when the pharma’s $4.8 billion acquisition on Nanobody developer Ablynx NV paid off following the FDA’s green light for Cablivi (caplacizumab-yhdp) in the rare and sometimes fatal blood disorder acquired thrombotic thrombocytopenic purpura (aTTP). (See BioWorld, Feb. 7, 2019.)
Cablivi is designed to work by neutralizing the large blood glycoprotein von Willebrand factor (vWF), which is involved in homeostasis and is critical for thrombus formation. In patients with aTTP, there is an accumulation of vWF that results in a formation of clots in the small blood vessels, causing severe thrombocytopenia, tissue ischemia and organ damage.
Second placed Abbvie Inc. is, once again, having a productive year in terms of new product approvals. In August, Abbvie Inc.’s oral rheumatoid arthritis (RA) drug, upadacitinib, won U.S. approval following a priority review at the FDA. (See BioWorld, Aug. 19, 2019.)
The potential blockbuster JAK1 inhibitor, to be marketed as Rinvoq, is indicated for adults with moderate to severe RA who have had an inadequate response or intolerance to methotrexate..
Rinvoq is Abbvie’s second targeted immunomodulator after Skyrizi (risankizumab), a medicine that won FDA approval for adults with moderate to severe plaque psoriasis in April. The company paid Boehringer Ingelheim GmbH $595 million up front for rights to the anti-IL-23 therapy and another asset in 2016. (See BioWorld, April 25, 2019.)
Although Novartis AG fell six places in this year’s index rankings, it is already making a strong case for a higher spot in the next index rankings, based on the number of new medicines that it has brought to market this year. In February, it won FDA approval for Egaten (triclabendazole) for fascioliasis, or liver-fluke disease, in patients 6 and older. A month later, its oral sphingosine 1-phosphate (S1P) receptor modulator, Mayzent (siponimod), was given the nod for adults with relapsing forms of multiple sclerosis. (See BioWorld, May 28, 2019.)
Its Avexis unit’s new gene therapy, Zolgensma (onasemnogene abeparvovec), won FDA approval for the treatment of all types of spinal muscular atrophy in children under 2 with biallelic mutations in the SMN1 gene. Closely following was the FDA approval of Piqray (alpelisib), the first PI3K inhibitor to be marketed for the treatment of breast cancer. (See BioWorld, May 28, 2019.)
Four other big biotechs achieved top 30 index rankings this year: Biogen Inc. (13), Amgen Inc. (15), Celgene Corp. (16) and Regeneron Pharmaceuticals Inc. (17). Both Biogen and Amgen dropped three spots from their 2018 positions, with Regeneron gaining three and Celgene jumping one position.
Amgen could move up next year as it already has had success with the FDA approval of its UCB SA-licensed sclerostin-neutralizing antibody, Evenity (romosozumab), for the treatment of osteoporosis in postmenopausal women at high risk of breaking a bone. (See BioWorld, April 10, 2019.)
The approval also came with a requirement of Amgen to run a five-year postmarketing study to assess the cardiovascular safety of the therapy, potentially followed by a comparative safety study or trial.