Replies to post #98 on PTC Therapeutics Inc (PTCT)

[girlfriend]

Thank you…..I am also wondering if there would be any reaction in sympathy to the wait and see approval for Serapta's drug of which the FDA recently requested more information of.

[chainma1l]

Would NHS have bothered with this MAA if NICE wasn't on board already?
EMA review could be announced at any time now. What happens if they say that the yearly review is no longer necessary? $20+?

8:19 am PTC Therapeutics negotiates a managed access agreement for Translama for ambulatory patients aged 5 years and older with nonsense mutation Duchenne muscular dystrophy (PTCT) :

Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation.
•This decision provides reimbursed patient access to Translarna in England via a five-year MAA.
•NICE is expected to issue final guidance later this month following execution of the MAA, with implementation soon after.
•PTC and NHS England have now finalized the outstanding aspects of the MAA which include a confidential financial arrangement and the collection of further data on the efficacy of Translarna for the treatment of nmDMD over a five-year period with NICE guidance to be reviewed again at the end of that period, before future funding decisions are taken.
•An opinion on renewal of Translama is expected mid-2016 by the EMA