Novartis bets big as RNAi reaches possible inflection point
Novartis is to acquire The Medicines Company for $9.7 billion and will gain access to the biotech's RNAi-based cardiovascular therapy inclisiran. Based on positive Phase III data, inclisiran is shortly expected to be submitted to regulators for approval.
The drug is designed to lower cholesterol levels in patients with atherosclerotic cardiovascular disease or a genetic disease that results in high amounts of cholesterol in the blood and is administered to patients just twice a year. As a result, Novartis hopes that improved convenience and superior compliance will allow inclisiran to compete against marketed biologic products that also target PCSK9 in order to lower cholesterol (which have largely failed to meet pre-launch expectations).
Acquisition of The Medicines Company is further evidence of RNAi's arrival; the FDA recently granted approval to a second RNAi therapy developed by Alnylam Pharmaceuticals and there has been a spate of dealmaking in this space, such as Novo Nordisk's recently announced cardio-metabolic collaboration with Dicerna. At its recent R&D day, Alnylam updated investors on its plans to expand RNAi drug development into central nervous system and ocular indications in partnership with Regeneron Pharmaceuticals.
The FDA shows no sign of slowing down as Oxbryta approved for sickle cell disease
On Monday, the FDA continued its recent run of approving novel, breakthrough drugs notably ahead of schedule by green-lighting Global Blood Therapeutics' Oxbryta for sickle cell disease (SCD).
The FDA had previously set a PDUFA date of February 26, with Global Blood Therapeutics having only had its new drug application accepted by the agency in early September. Oxbryta is the second new SCD treatment approved for the US market in a matter of weeks. Novartis' Adakveo was authorised earlier this month.
Over the past month, the FDA has approved five new therapies - each costing in excess of $100,000 a year - months earlier than expected, indicating that the agency is clearly unperturbed by suggestions it sometimes acts too hastily in facilitating market access for new drugs.
Amarin's Vascepa up next
The FDA has a number of other notable regulatory decisions to make before 2019 draws to a close, the most significant of which is whether to expand labelling for Amarin's purified fish oil drug Vascepa to include prevention of cardiovascular events. Doing so would potentially set the drug up as a multi-billion dollar product.
A unanimous AdCom recommendation in favour of Vascepa earlier this month bodes well for Amarin; we recently spoke to the company's CEO John Thero about what comes next.
Risdiplam poised to be a 2020 regulatory highlight
Roche set up what will be one of the most eagerly awaited new drug approvals in 2020 this week by confirming that the FDA has granted priority review to its application for risdiplam, an experimental treatment for spinal muscular atrophy (SMA)
Expectations are high as risdiplam is not only on track to be the third SMA treatment to reach the market in little over three years, but will be the first oral agent. It has also demonstrated promising data in older patients, which could provide further differentiation versus competitors.
Keytruda misses out on NRDL status
Chinese regulators announced Thursday that 70 new medicines have been added to the country's National Reimbursement Drug List (NRDL), with companies agreeing to cut prices by an average of 61%. The National Healthcare Security Administration (NHSA) said that the therapies added to the list include AbbVie's TNF inhibitor Humira (adalimumab), Roche's breast and lung cancer treatments Perjeta and Alecensa, as well as AstraZeneca's roxadustat; which has been approved in China ahead of western markets. The NHSA added that almost all of the imported medicines newly added to the list had the "lowest prices globally."
A notable exception is Merck & Co.'s PD-1 inhibitor Keytruda, potential inclusion of which on the NRDL had been watched closely by investors in recent weeks. Instead Tyvyt, which was developed by Innovent Biologics and Eli Lilly, and is approved in China for the treatment of relapsed or refractory classic Hodgkin's lymphoma, is the first PD-1 to be included on the NRDL.
To read more Friday Five articles, click here.