by Raphaël Moreaux
BASEL, 6 Sep (APM) - Almost 75% of Novartis' R&D projects in 2019 are focused on ''first-in-class'' drugs, according to data presented at a press visit to the group's headquarters in Basel, Switzerland.
Novartis' pipeline has 30 new drugs across 45 diseases and the company invests $4.6 billion annually into all the therapeutic areas covered by the pharma (oncology, immune-oncology, respiratory, ophthalmology, neurology, cardiovascular diseases, infectious diseases, immunology, hepatology and dermatology). It is currently conducting more than 500 trials involving more than 80,000 patients.
At the moment, 73% of projects in development concern potential first-in-class drugs and 18% are first-in-class for the targeted indication.
Novartis is intending to create new standards in oncology, which makes up 40% of its R&D activity, by combining several innovative therapeutic approaches such as targeted therapies, immunotherapies, CAR-T and radioligands, said Global Translational Medicine Head, Musculoskeletal Diseases at Novartis, Ronenn Roubenoff.
He emphasised that Novartis has a wide range of potential new drugs to treat diseases for which there is currently no medical response.
One example is TQJ230 (with Ionis Pharmaceuticals - APMHE 62010
) being evaluated to reduce risk of cardiovascular disease in patients with elevated lipoprotein(a) (Lp(a)) and ApoCIII. Another is long-sightedness treatment UNR844, from the 2016 acquisition of U.S. company Encore Vision (APMHE 50989
). Cell and gene therapies are also being developed (APMHE 64255
By the end of 2019, Novartis is counting on having had more than 26 marketing authorisations and opinions from regulatory agencies worldwide.
One of its most innovative treatments, gene therapy Zolgensma (onasemnogene abeparvovec), was approved in May in the U.S. for paediatric patients under two2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the SMN1 gene (APMHE 63120
). A European approval decision for the product, which is the most expensive in the world with a catalogue price of $2.125 million for a single injection, is expected in the second half of 2019.
Novartis is expecting to file the drug for approval for SMA types 2 and 3 in 2020.
European approvals or opinions are also expected before the end of the year for indication extensions in Lucentis (ranibizumab) for diabetic retinopathy and retinopathy of prematurity and Mayzent (siponimod) in secondary progressive multiple sclerosis. The latter, a potential successor to blockbuster Gilenya (fingolimod), was approved in the U.S. in March.
Novartis is also expecting a regulatory decision in the U.S. before the end of the year for brolucizumab in the wet form of age-related macular degeneration (AMD).
Elsewhere, the pharma is expecting to have filed for approval in the U.S., Europe and Japan for its immunology treatment Cosentyx (secukinumab) in non-radiographic axial spondyloarthritis (nrAxSpA) and Entresto (valsartan+sacubitril) for heart failure with preserved ejection fraction. In this indication, the drug received negative results in Phase III, but a subgroup analysis suggests there is a benefit for some patients (APMHE 63855
, APMHE 64212
Cosentyx and Entresto are two of Novartis' main growth drivers. The first is currently approved for psoriasis, psoriatic arthritis and spondyloarthritis. The latter is approved for heart failure with reduced ejection fraction.
Another drug awaiting approval for expanded use is Xolair (omalizumab) for nasal polyposis. It is already approved for allergy-related asthma and idiopathic spontaneous urticaria.
Novartis will file for initial approval in the U.S. and Europe in 2019 for ex-PDR001 (spartalizumab) in combination with Tafinlar (dabrafenib) and Mekinist (trametinib) in metastatic melanoma with BRAF V600 mutation and for Arzerra (ofatumumab, with Genmab) for recurrent multiple sclerosis. It will file for approval in the U.S. and Japan for INC280 (capmatinib) for non-small cell lung cancer (NSCLC).
Novartis is planning to file for approval for two new drugs in 2020: QAW039 (fevipiprant) for asthma and LU-psma-617 Radioligand therapy for castration-resistant metastatic prostate cancer. It is also planning to file for approval for Entresto for expanded use as post-acute myocardial infarction.