ACADIA TALKS TROFINETIDE, page-67

Sillazze

I would love that to be the case but….. I don’t think this is comparing apples with apples.

Keselugo is for a disfiguring, painful, inoperable paediatric disease which causes tumours to grow on nerves (that can weigh up to 20% of body weight) and for which there is no current treatment.

Results were quite significant - two thirds of patients in the trial had a confirmed partial response (tumour shrinkage) and 80%+ maintained that response for more than a year. Also, the drug had a significant impact on pain to the degree that patients who had been suffering chronic, debilitating pain came off pain medications.

Keselugo received Breakthrough Therapy Designation last year.

The US government provided assistance with the trial.

Trial results were published in the prestigious New England Journal of Medicine.


One interesting fact I noted is that, since first licensing this drug from Array Biopharma in 2003, Astra Zeneca had attempted development in several oncology indications only to endure a string of trial failures.

I think it’s fantastic to see that AZ’s persistence has been rewarded and even more fantastic that there is now a treatment which can provide some help to the poor kids born with this condition.

I’m hoping that Neuren and Rett families will be similarly rewarded.

https://1businessworld.com/2020/04/...gical-disorder-after-17-years-of-development/

https://oncologynews.com.au/selumet...vides-clinical-benefit-for-children-with-nf1/