The UK drug regulator has granted volanesorsen a positive scientific opinion under its early access to medicines scheme for use in patients with familial chylomicronemia syndrome. The drug’s sponsor, Akcea Therapeutics, is hoping to get the drug approved in the EU, the US and Canada this year.
Akcea Therapeutics has received permission to make its unlicensed treatment for familial chylomicronemia syndrome (FCS) available to patients in the UK under the government’s early access to medicines scheme (EAMS) while the product undergoes evaluation for marketing throughout the EU.
Volanesorsen, an EU orphan designated drug for treating patients with the rare lipid disorder, was granted a positive scientific...
Despite pushback by industry, the government is standing by its new statutory scheme rebate rates for branded medicines, including one that doubles the rate that companies must repay on the sales of newer products to the National Health Service to 31.3%.
Companies operating in Argentina may get their medicines to the market more quickly thanks to new updates to the medicines authorization system.
While the adoption of most favored nation drug pricing in the US stands to affect Japanese biopharma firms now heavily reliant on this market, it might also present an opportunity for pricing and policy reforms at home.
European health systems already pay far too much for new medicines and payers will not accept higher prices to compensate for lower US prices, according to Anja Schiel, from NOMA, the Norwegian health technology assessment body.
