David Vaczek speaks with PTC’s Mark Rothera about the global launch of its lead product and how the biotech is gearing up to commercialize Duchenne drug Translarna in the US. The following is edited from a longer discussion and is part of an occasional series of interviews with biotech marketers.

DV: Translarna (ataluren) is in Phase III in this country but is currently launching overseas. With the drug now available to some patients in Europe, what are the next steps for commercialization?

MR: The launch in Germany is an important step because not only is it the first time we are launching Translarna around the world, but also the first-ever drug to be specifically launched for Duchenne muscular dystrophy (DMD). We are pursuing two parallel tracks. The gating event for commercialization is the pricing and reimbursement negotiations with the EU states which can take anywhere from three to 18 months. Going into 2015, we are pursuing access to the drug in Sweden, Denmark and Norway, and in the second half of Q2, the UK, followed by France, Italy and Spain.

We feel equally passionate about early access programs, where countries allow reimbursed drug before formal commercial launch for rare diseases for which there are no therapeutic alternatives. Boys with this severe muscle-wasting disease have a life expectancy in the 20s, so every day counts. We are already providing reimbursed drug into France, Spain and Italy, and now Turkey and Israel.

DV: How is Translarna being marketed to HCPs and patient groups?

Rothera: To have a first drug that has made it over the line with regulatory approval is a huge moment for the DMD community and has given them a lot of hope for the future. One of the challenges is helping them to understand the realities of the market-access process. We are focusing on patient genotyping to make sure the right patients–the 13% of DMD patients with a nonsense mutation–are accurately identified as right for the drug. As no therapy has been available, not all patients have been genotyped. Patient groups are playing an active role here. They are helping with compliance education, an important aspect for a new oral therapy taken three times a day by children of school age.

We are putting on master classes at support centers where physicians can hear from experts on best practices and what holistic management of the patients is like. The number of centers of excellence can be quite limited for rare diseases, and you find physician competence varies. You can help improve standards along with the community.

DV: What will Translarna cost?

MR: Ultra orphan therapies are typically priced in the $200,000 to $400,000 a year range. This type of therapy is 100% reimbursed in the EU through agreements reached at the country level based on price and how many patients will benefit.

DV:  What is the efficacy bar for the ACT DMD confirmatory Phase-III trial, for supporting full EU approval, and the year-end NDA rolling submission filing for Translarna with FDA?

MR: We learned in our Phase-2b study which patients we will need to select for the confirmatory trial to be able to show the effect of the drug most clearly in a 48-week period. We showed an average 30-meter difference in walking ability in Phase 2b between active and placebo arms. We expect to show at least that because of the improved selection. The trial will read out [this] year in the third quarter when we would expect to then use it as the basis for completing our submission to FDA.

DV: Prosensa and Sarepta target a different kind of DMD genetic mutation. How will they impact the landscape should they come to market?

MR: There is no overlap between their drugs and ours as they treat different subpopulations with DMD. All the drugs have benefitted from learning on the progression of the disease with these mutations.

DV:  PTC is pursuing indications for cystic fibrosis and MPS1. How big is Translarna’s potential?

MR: Translarna is a kind of proof of principle for treating diseases caused by a nonsense mutation, which is present in up to 2,000 monogenic diseases, with about 11% occurance on average. So DMD could be just the beginning in changing the courses of these diseases. It’s been a 16-year journey to this point. We have had to pioneer getting a first-in-class drug with a novel mechanism of action to market. We were the first to pioneer a clinical development end point for regulatory purposes, and the first to go through the regulatory process to a conclusion. Along the way, we have had to learn and share that learning with a lot of people and communities.