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Expert Interview

Slingshot members are talking to an expert! The topic is:

A look at the use of Tyvaso (treprostinil) in PAH and the potential of Tyvaso DPI, in light of its delayed FDA review due to lung safety concerns

Ticker(s): UTHR

Who's the expert?

Institution: UNC Chapel Hill

  • Associate Professor of Medicine and Director of Pulmonary Hypertension Program at UNC Chapel Hill where he manages treatment for more than 350 patients with PAH.
  • Interested in clinical and translational research in the area of Pulmonary Arterial Hypertension (PAH).
  • Involved in numerous multicenter clinical trials investigating therapies for PAH and investigator initiated studies examining the treatment of PAH in the setting of sarcoidosis, cirrhosis, and interstitial lung disease (pulmonary fibrosis) as well as research investigating the role of inflammatory and procoagulant biomarkers in the pathogenesis and clinical manifestations of PAH.

Interview Questions
Q1.

Please describe your specialty and details about your practice

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Q2.

How many patients do you see with Pulmonary arterial hypertension and specifically with pulmonary hypertension with Interstitial Lung Disease (PH-ILD)?

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Q3.

For PAH, how has your use of Tyvaso changed with the evolving treatment landscape and with other formulations of generic treprostinil approved for PAH?

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Q4.

In terms of Pulmonary hypertension with ILD, can you describe how you treat these patients, what are the options and where Tyvaso fits into that?
Given it is the only FDA approved treatment with this specific indication, since its approval in 2021, has it become the standard and your preferred option?  

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Q5.

After their approval in May 2021, although subcutaneous and IV generic treprostinil are approved only for PAH, and not specifically PH-ILD, Is there any use of subQ or IV forms of treprostinil off label for PH-ILD?

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Q6.

Are these and other generic formulations expected to work for PH-ILD or do you see them as inferior options? Or does the FDA label indication drive your prescribing preference?

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Q7.

Patient experience with Tyvaso:

How has it been working in your hands?  
Any feedback from patients?

Any key things you are seeing in terms of safety? 

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Q8.

Accessibility and affordability for patients?

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Q9.

If you had to rate your level of excitement about Tyvaso for PH-ILD on a scale of 1-10 what number would you choose and why?

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Q10.

Any other important points to be made on Tyvaso that we haven't touched upon that you want to elaborate on?

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Q11.

Potential of Tyvaso Dry Powder Inhaler (DPI) in PAH and PH-ILD
Please discuss the results of the BREEZE trial and how you interpreted that.

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Q12.

Please comment on the findings of the BREEZE study:
-Significant improvements in Patient satisfaction with the device (Preference Questionnaire for Inhaled Treprostinil Devices) (PH-ITD)
96% agree or strongly agree to overall satisfaction with the DPI at 3 weeks compared to 31% agree/strongly agree at baseline with Tyvaso (nebulizer) and 45% neutral. 
-Significant improvements in 6MWD
-PAH SYMPACT scores trend of improvement and stat sig on physical impacts score at week 3 and 11, stat sig on cognitive/emotional impacts score at week 3, trend at week 11. 


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Q13.

Why might 6MWD improve going from Nebulizer at baseline to DPI? Is this effect real?

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Q14.

How do you see the market opportunity for Tyvaso DPI in PAH and PH-ILD?  Would you switch patients from Tyvaso to Tyvaso DPI, and would DPI expand the market?

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Q15.

A Citizen's Petition to the FDA has delayed approval of Tyvaso DPI, with the PDUFA now pushed to this month.  This was due to an excipient in the formulation. Mannkind's dry powder inhaled insulin product AFREZZA has this excipient, and this product has a black box warning due to potential for bronchospasm in patients with lung disease. So, is this potential side effect a concern and would a black box warning concern you or impact your use of Tyvaso DPI if approved?   Do you expect Tyvaso DPI to be approved?  

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Q16.

Yutrepia/competition
There is some speculation the citizen's petition was written on behalf of Liquidia which has a competing DPI, Yutrepia, now tentatively approved and stayed pending the expiry of exclusivity of Tyvaso.  Are you familiar with Yutrepia, and do you see any differentiation? 
It does not contain the same excipient so would it make a difference if there is no black box, in choosing between these products?  Would the first to market advantage be more important in PH-ILD and therefore Tyvavso DPI with a head start could still take majority share?  

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