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June 8, 2017: The case for early approval of Voclosporin

Voclosporin will likely be approved well before the P3 trial runs it course.

Here's how it's going to work. 

First, there's a legal and regulatory path for this approval.  While I quote the relevant passage below, here's the link to the FDA Regulations, as well as a list of drugs that received accelerated approval. 

Sec. 314.510 Approval based on a surrogate endpoint or on an effect on a clinical endpoint other than survival or irreversible morbidity.

FDA may grant marketing approval for a new drug product on the basis of adequate and well-controlled clinical trials establishing that the drug product has an effect on a surrogate endpoint that is reasonably likely, based on epidemiologic, therapeutic, pathophysiologic, or other evidence, to predict clinical benefit or on the basis of an effect on a clinical endpoint other than survival or irreversible morbidity. Approval under this section will be subject to the requirement that the applicant study the drug further, to verify and describe its clinical benefit, where there is uncertainty as to the relation of the surrogate endpoint to clinical benefit, or of the observed clinical benefit to ultimate outcome. Postmarketing studies would usually be studies already underway. When required to be conducted, such studies must also be adequate and well-controlled. The applicant shall carry out any such studies with due diligence.

Second, is a "surrogate endpoint?"   According to the FDA, surrogate endpoint is "a laboratory measure or a physical sign that is intended to be used as a substitute for a clinically meaningful endpoint."  There are two relevant surrogate endpoints for Voclosporin, and the first is UPCR.   Per a National Institutes of Health document,  "Proteinuria is a major predictor for progression of renal disease, including diabetic nephropathy. In a post hoc analysis of the ORIENT, a double-blinded randomized trial of 566 type 2 diabetic patients with nephropathy, we examined the risk association of composite renal outcome [end-stage renal disease, ESRD, doubling of serum creatinine (SCr) and death] with baseline, change and residual urinary protein/creatinine ratio (UPCR)."   Soooo, if the UPCR is falling a rate consistent with with the P2 trial, that indicate you're route to a positive clinical outcome.

Now add in the SLEDIA Index (Systemic Lupus Erythematosus Disease Activity Index), which is "a list of 24 items, 16 of which are clinical items such as seizure, psychosis, organic brain syndrome, visual disturbance, other neurological problems, hair loss, new rash, muscle weakness, arthritis, blood vessel inflammation, mouth sores, chest pain worse with deep breathing and manifestations of pleurisy and/or pericarditis and fever."  See 

Punch line: If UPCR and SLEDAI are on track and consistent with Phase 2 data with similar P-Values, it is entirely possible that Vocloporin receives accelerated approval.  If that happens, the P3 trial would continue,  and additional data would be collected during the marketing phase to assure the decision to market is supported by robust clinical evidence. 

Comments

  1. Other than Auphenia, are there any groups advocating for an early approval including the SLE support organizations? One would think that the LN patients themselves would be pushing the FDA to get the drug approved ASAP. A small company like AUPH doesn't have much clout with the FDA the way BP has. Perhaps some of the influential investors can put in a good word with their FDA buddies on the 19th hole.

    ReplyDelete
    Replies
    1. The Lupus Foundation has been outspoken, mentioning Aurinia and Voclosporin multiple times in their official publications, including a description, recently, of the Madrid paper. The Lupus Foundation has a national chapter with lobbies Congress, and many local chapters across the country. I know that the National Chapter of the Lupus foundation is already engaged at a Congressional level on Lupus issues generally, and had recently legislative win securing federal funding for Lupus related research. Moving forward, I believe the National Chapter will be coordinating advocacy work to encourage the FDA to expedite approval of Voclosporin on behalf of the millions of lupus patients they represent across the country. We've not yet discussed the potential impact of the Lupus Foundation's National Chapter, but I believe it could potentially be substantial

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    2. The fact that these groups have been so active in trying to get Voclosporin approved is great news. Aurinia (did I actually say Auphinia earlier?) has limited resources for PR as a small company so obviously the push to get early approval has to come from advocacy groups such as the ones you mentioned. A more receptive FDA may be the key to getting the drug approved earlier.

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  2. Vid, somehow someone mentioned in the IHUB board that the New FDA head (Gottlieb?) has had some connection with Aurinia like he was a shareholder and another guy responded to that post saying that this guy just have to sell his holding if he is to assume that position as head of the FDA. I was wondering if you came across those posts.

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    Replies
    1. Yes Jess and that was exactly what I was alluding to when I said "a more receptive FDA". It cant hurt having him as head of FDA. Even if he sells his position you can rest assured how he feels about AUPH.

      Delete
    2. Vid, one poster at IHUB responded to another saying"the only thing here you will hear outside of BR is crickets", lol🤣🤣🤣

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    3. Jess, Gotliebb was a partner at NEA, one of Auph's largest holders. He has removed himself from decisions that have ties to his investments for just a 12 month period.

      Delete
    4. I've not heard of iHub. Is that a car part or something??

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    5. If BR ends up posting to himself on liehub, can he get his admin buddies to delete his posts? If a tree falls in the forest with no one around, does it make a noise?

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  3. TC-

    You should edit the settings of this blog so that people can subscribe to receive emails about new posts. If you are interested in setting this up you can go onto the main blogger homepage, click on Layout, then Subscription, then Follow by Email, Show Follow by Email.

    Would be a nice touch.

    -John

    ReplyDelete
    Replies
    1. Excellent suggestion, john. Let's see if I can set that up tomorrow (after I recover from binging on House of Cards).

      Thanks!

      Delete
    2. Subscriptions now working! Thanks again, John.

      John (aka TC)

      Delete
    3. Awesome! I will subscribe now!

      Delete
    4. Great work . Congrats to both of you. Just subscribed....will make it very easy for anyone to track down new posts. Another nail in the Liehub coffin hopefully.

      Delete
    5. Hi, you can also follow using RSS feeds:

      Posts:
      http://theauphians.blogspot.com/feeds/posts/default?alt=rss

      Comments:
      http://theauphians.blogspot.com/feeds/comments/default?alt=rss

      Hello to everyone btw :)
      --
      Bogusz

      Delete

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iHub has been hijacked by trolls

Yup. iHub can't get rid of "Biotech Researcher," aka BR.  It's both a win for BR (the troll), and a huge loss for iHub. Their platform loses value and credibility when they refuse to take action on perma-trolls that derail the discussion and distract investors from their efforts to just honestly share information and opinions on the equities they own.

So shame on iHub for not taking action to resolve this.

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With kind regards to all

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