PTC-Duchenne Advocacy Community Call, March 21, 2017

Here is a summary of today’s call with PTC’s Patient Advocacy and Commercial Teams

Thank you PTC for hosting a call for advocacy leaders this morning to introduce the EMFLAZA team and give us a preview of the upcoming launch of this drug.

PTC is committed to bringing treatments to rare diseases where there are no, or few treatment options.  EMFLAZA is underutilized amongst Duchenne patients across the country and they are dedicated to providing access to all patients.  EMFLAZA is a complimentary therapy to Translarna and they will have systems in place to provide both therapies to the community.

PTC plans on closing the acquisition from Marathon in mid-May.   They are in the process of collecting prescription and start forms from Marathon which will be filled after the transaction is closed.

The company is not able to comment on pricing at this time due to anti-trust and regulatory issues.  They are already engaging with payers, starting with commercial payers first.  Each patient that has submitted a start form will be communicated individually on a case-by-case basis.  Case managers will be assigned to each patient and will help ensure access during the transition.  The case managers will work with the uninsured and direct patients to organizations which will be able to provide the drug, if necessary.

PTC will continue to communicate with Duchenne organizations and they are working on a letter to post to the broader community.

In regard to Becker patients and other indications, they are working with Marathon on establishing standard operating procedures (SOP).

During this transition time, patients will be able to order from Masters or their current supplier.  At the time of commercialization, FDA regulations do specify that importation is not possible.  Masters UK has indicated it will no longer ship to the US after commercialization, PTC will meet individually with families who have concerns about transitioning to EMFLAZA.

Concerns were voiced about the ability, willingness or wisdom of having multiple high-priced drugs that need to be taken in combination, and the burden it will place on the health care system.  PTC acknowledges this is the time to re-examine the strategy.  Examples were given about other diseases, such as cystic fibrosis, that evolved from no treatment, to one treatment, to multiple treatments.  PTC feels that insurers are aware of this model and that they will understand the value of combination drugs that slow the progression of disease.

In summary, the main issue of pricing was not discussed due to regulatory mandates.  PTC promised to communicate regularly during this process.  We appreciate PTC’s dedication to the Duchenne community and we look forward to ongoing two-way dialog with the company.