World Orphan Drug Congress LIVE: Commercialization

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Back in our main conference room, our commercialization segment of the afternoon began. Mark Rothera Chief Commercial Officer of PTC Therapeutics spoke about launching the 1st therapy for DMD- a 16 year journey! Here are some of the points he touched on,

  • Collaborating with the expert community and international patient groups from ‘R’ to ‘D’ to ‘C’
  • Going it alone or partnering for launch?
  • The importance of early and expanded access programs
  • Benefits of genotyping with the advent of therapy

PTC Therapeutics is committed to developing treatments for rare disorders with a life-threatening unmet medical need. During the presentation, Mark Rothera explains post-transcriptional control- platform technologies including:

1. Nonsense Readthrough

2. Alternative Splicing

3. Nucleotide repeat

4. Protein Modification

5. Transcript Regulators

PTC has persevered through unchartered territory in drug discovery, clinical development and now commercializing 1st drug for nmDMD. The Phase 2b placebo-control study data defined natural history of 6MWD. 6MWT outcomes with Translarna compare favorably with those for other approved drugs for rare disorders.

PTC and Patient Advocacy groups have partnered to provide treatment for DMD patients.

Mark also addressed the importance of early and expanded access programs. Translarna’s access is expanding rapidly, focusing first on fastest access geographic areas.

A precision medicine for a rare disorder has to be delivered to the right patients. PTC is tackling the issues, which include late diagnosis, lack of knowledge of genotyping, lack of genetic testing, or the patient is outside of a major center.

Kate Holland, VP, Commercial, Vanda Pharmaceuticals gave a fantastic presentation on the grassroots method of achieving expanded disease awareness and commercial success without a patient group. Some of the points Kate touched up on included:

  • How traditional marketing early in the process can help raise awareness for your rare disease
  • Empowering your patients to grow “patient-directed physician visits”
  • Overcoming the knowledge gap and incorrect diagnosis of your disease

Kate discusses Vanda Pharmaceuticals tactics in addressing the issue of Non-24- a serious circadian rhythm disorder. She quotes, “It’s all about the patien.” It’s about what the patient voices out, what types of channels they would like to be reached by based on their abilities. Vanda Pharmaceuticals used radio and television as a result of listening to people and finding out how they would like to be reached. They threw an innovative approach to an orphan indication, which revolved around the patient and the patient journey.

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