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Expert Interview

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

Discussing the potential for US FDA approval for Soliris (Eculizumab) in treating Refractory generalized myasthenia gravis before FDA decision expected Oct. 23, 2017

Ticker(s): ALXN

Who's the expert?

A neurologist with experience in treating Myasthenia gravis or other neuromuscular diseases.

Interview Questions
Q1.

Please describe your clinical practice. How many patients with Myasthenia gravis do you currently treat?

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Q2.

Can you talk about the patient populations suffering from Myasthenia gravis. Does the onset happen only past 40-60 years old? Once damage to the Nicotinic receptors has occurred, is it reversible, or rather, are the treatment options viable only for preventing the worsening of the disease?

Added By: c_admin
Q3.

In its other indications for treating  paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, Eculizumab increases the risk for meningococcal infections by up to 2,000 times, and as such, Eculizumab is available only through a restricted program.

  • From your experience, does this greatly impede patients access to this medication? Are there safer alternatives for myasthenia gravis? 
  • In your opinion is it a risk doctors and patients would be willing to take?

Added By: c_admin
Q4.

Eculizumab in the PNH indication costs around ~$400.000/year.  How much do alternatives in treating Myasthenia gravis cost at the moment. What hurdles does the drug have to overcome regarding payor reimbursement?

Added By: c_admin
Q5.

Phase 3 participants achieved a Myasthenia Gravis Composite (MGC) p-value of 0.1026 at week 26, change from baseline at week 26 being 0.0406. 

  • What is your general opinion of the P3 data?
  • If approved in the US, how likely would you be to switch patients off of existing first-line therapies based on this data?

Added By: c_admin
Q6.

On a scale of 1-10, 10 being very excited, how would you rate your level of excitement for this drug?

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