Theralase Technologies Inc. V.TLT

Preliminary Breakthrough Therapy Designation (BTD) Advice

[Eoganacht]

The FDA BTD FAQ site makes it clear that preliminary BTD advice is advisory and does not predict the actual BTD decision.

Frequently Asked Questions: Breakthrough Therapies

Question 22. What are the timelines for FDA to respond to a breakthrough therapy designation request? 

 

FDA will respond to breakthrough therapy designation requests within 60 days of receipt of the request. 

Question 23. Can a sponsor get preliminary breakthrough therapy designation (BTD) advice from the review division prior to the submission of a formal BTD request?

 

A sponsor can contact the regulatory project manager (RPM) in the division to which the active IND is assigned and request the “Preliminary Breakthrough Therapy Designation (BTD) Advice Request” template. This template should then be submitted as a formal amendment to the IND and a subsequent teleconference between the sponsor and the review division will be set-up by the RPM. The review division will make a recommendation as to whether a request for a BTD is appropriate, may be too preliminary, or does not currently meet the criteria for a BTD. The Agency’s recommendation is advisory and is not to be interpreted to predict the Agency’s decision on the BTD request. NOTE: A Preliminary BTD Advice Request may be submitted to an active PIND, although a formal BTD request may not be submitted until the IND is opened.

[Maxcitrac]

Very good point Eoganacht, I like the highlited part that say's  or does not currently meet the criteria for BTD. Please correct me if I am wrong, but what it does imply is, if all the necessary criterias for the BTD approval are not included in the Pre-BTD application,TLT could have to refile a new Pre-BTD application with the missing informations. My point is if all the criterias for

[Eoganacht]

Hi Maxcitrac - preliminary BTD advice is a useful tool but it's up to trial sponsors whether they want to use it or not, although it's hard to imagine why a sponsor would not take advantage of it.

[wildbird1]

Eoganacht, thanks for the clarification... At first glance Maxcitrac saying that after the Pre-BTD approval, the BTD approval is almost 100% guarantees, looks a little too much. But! If first we looks at these 6 facts about TLT-Ruvidar... 1)TLT-Ruvidar is the only ''one and done'' treatment in the world with the highest CR% in the class, that treat BCG resistant patients. 2)TLT

[Eoganacht]

Hi wildbird1 - like you I am convinced Ruvidar PDT will soon  get BTD - for all the reasons you cite. Now that Theralase has complied with the FDA recommendations it should (hopefully) just be a matter of crossing all the t's and dotting all the i's! 

[Dreimer200]

Wildbird1:  All the elements you adduce suggest why receipt of pre-BTD approval is material news – and should be communicated by the company to shareholders.

[menoalittle]

Which is also why nothing on it should be disclosed or released until after closing the PP... But after the PP... buckle up.

[Oilminerdeluxe]

Yes, that needs to close first, I think. So let's hope that happens fast! 

[chry200030]

When do we expect the PP to close. Any guesses? 

[TriumphSpitSix]

My understanding is that FDA Pre-BTD communications are "advisory" which simply means the FDA review division agrees (or not) that the company's pre-submission materials and data meets some minimum threshhold to move forward with an actual application for BTD. Think of it like a checklist for gathering documents and information when applying for a mortgage. Completing your checklist

[Dreimer200]

There are occasions when common sense should prevail over technicalities. Providing the news of pre-BTD approval would help to suggest the company is actually interested in keeping shareholders informed.    While the news does not, of course, guarantee ultimate approval – it does suggest that a significant hurdle towards that end has been removed.

[enriquesuave]

Basically it seems that if the FDA gives a thumbs up for BTD application, then it's a good sign but no guarantees.  If they don't agree, then you can still file BTD, but good luck.  I think we should fall in the first category.    

[CancerSlayer]

The pre-BTD option certainly fine tunes the process & helps minimze the possibility of having to re-submit a BTD request in the event of a first-time denial (i.e. due to inadequate number of patients reported, inadequate data to support "preliminary clinical evidence").  This preliminary option takes a big burden off of the FDA & sponsor...would hate to have to file more