The bottom line is that the meeting was not given the importance by the FDA so they were designated Type C. Your point is correct and that's why I said before that lot of small company CEOs think they are clever and can fool the FDA, naïve investors by using statements that appear very critical and important! the FDA is a veteran at this and they see through all these gimmicks! Lot of companies like these submit grandious meeting requests to the FDA and sometimes they are so outrageous that the meetings are denied outright! These small companies use these FDA meetings as propaganda to lure naïve investors!! Recently, SRPT CEO used a small exploratory 12 patient trial and demanded an accelerated approval from the FDA. he was making all kinds of press statements for over a year prior to the FDA meeting and until the FDA meeting outcomes were released and then the stock crashed from 50 to 13 and now recovered to 18.
Then the 8k filing needs to be updated or revised. It is misleading because 1) BTD is not an approval pathway only accelerated approval is in that statement from CEO. 2) Type C meeting is not a critical regulatory milestone meeting. That's why it is conducted after 75 days or longer from the company's request date. Versus, Type B is conducted with 60 days and Type A in 30 days.
I don't know for sure the specifics but because of my experience observing theses several small company gimmicks, the answer is very simple:
From my industry regulatory experience, I know for sure that the management is already aware of the meeting outcome. Like I said before, the critical outcome details will never change from what was concluded at the end of the meeting on December 16th Vs what one would receive in 30 days. So when the CEO said he is waiting for official meeting minutes in 30 days, I know that he is delaying the news for 30 days. Bottomline, the CEO will not wait for 30 days to release if it was a good news! In fact the Oncology Division wont wait for full 30 days to issue the final minutes, it may come within two weeks if the company pushes for it because there will not be much difference. FYI, the meeting minutes is basically FDA responses to company's questions raised in a Type C meeting package. The preliminary responses are already known three days before December 16th. Secondly, the FDA will never agree in a Type C meeting that a drug could be filed via accelerated approval. Thirdly, the approval of Yervoy and Roches drug, will preclude any drug from getting accelerated approval using surrogate endpoints for Melanoma. Currently you need Overall survival data in a phase III trial. Since the CEO used BTD (breakthrough designation) and referred to it as one of the approval pathways, he is either completely ignorant or deliberately mislead the investors by using the currently famous phrase, which is "BreakThrough Designation" as an attention seeking gimmick so naïve investors will start buying the stock!
The only meetings that fit with NDA/BLA or even filing any key regulatory milestone event (fyi-IND filing, NDA filing etc are regulatory milestones) is Type B. Type As are usually reserved for removing bottlenecks developed during the key drug development process steps like getting an agreement for phase III protocol using SPA (Special protocol Assessment) prior to trial start or lifting a clinical hold on a trial etc.
So Type C is usually for non-key regulatory milestones meetings like getting advice on, can we get a Fast-track designation for our drug or BTD, or simply how do we (or need to) conduct nonclinical studies to support an eventual NDA or what kind of clinical studies needed for an eventual NDA or CMC related, mostly getting advice before conducting non critical tasks, which nevertheless needed for an EVENTUAL NDA. as opposed to critical task such as pivotal phase III trial or lifting clinical hold due to toxicity....
Sorry for the long answer. Hope this helps. I've seen lot of CEOs who claim they have qualifications/experience like having a PhD next to their name think they can fool the naïve investors. Look at the CEO's bio he claims he has regulatory affairs experience but I can tell you he has never brought a product (drug/biologic) to the market. or led a FDA meeting before he became a CEO....No schools teach drug development it comes only via actual experience. PhDs/MDs provide fundamental science knowledge but will not teach how to move a drug from phase 1 to phase 2 ot pjhase III transition from phase II or III to market etc......or how to conduct a GCP complient clin trials etc....
Basically the FDA probably said NO and that they need phase III trial for approval not phase 2 supporting accelerated approval, which the company met FDA to seek.!
"There are different possible routes to approval of PV-10 such as a breakthrough therapy designation or accelerated approval, and each of these has different requirements and time lines."
The above is totally misleading and incorrect! I am surprised a person at CEO level is making this statement. At least he could have consulted an industry experienced regulatory expert (not those CROs or who claim as consultants looking good on paper like having PhDs/MDs but no actual experience of having led several FDA meetings or approvals)
First of all, Breakthrough Designation is similar to Fast Track Designation which provides certain incentives during drug development and at filing stage. Accelerated approval falls within these two broad regulatory domains of BT or FT designations.
BT or FT are not approval pathways as he publicly claims. Accelerated approval is a pathway. Type C meetings are usually reserved for unimportant issues. Secondly, the meeting outcome is already known to the executives in a summary form. The 30-days is only a formality but the critical outcomes will not differ from what is known right after the meeting i.e. December 16th or 30 days later or sooner sometime 2weeks, when the document is released to the company. I don't understand why these small company CEOs think they are so clever just like AVEO, SRPT,etc.......and keep the bad news under wraps for 30 days. I am assuming that the bad news is that FDA is not willing to allow PV drug to be filed under accelerated approval for melanoma, even if the drug is designated either as BT or FT.
Any pharmaceutical company is obligated to submit the final study results from a clinical study within one year of completion per Code of Federal Regulations (No choice) but can submit earlier if FDA requests it. Please don't start misleading the naïve investors with your conspiracy theories.
One clarification: "FDA is obligated to release the official minutes TO THE COMPANY/SPONSOR within 30 days"
The disclosure of the content of the minutes to the public is only left up to the company not FDA per our US law passed by the Congress long time ago!
Let you and SRPT worry about the law suit recently filed against SRPT for misleading the investors....."Federman & Sherwood Investigates Sarepta Therapeutics, Inc. for Possible Securities Laws Violations"
FDA will only notify the adverse action to the company but no right to disclose that adverse action on an IND to the public......is this clear???
This law is especially relevant to the investigational drugs and pre-approval stage! For example, if a new IND is submitted to the FDA, FDA cannot disclose this fact. Only the company has the obligation whether or not to disclose! Well to answer your point, FDA has many regulatory actions to take if a company does not submit data to IND. They may put a hold on the IND etc.....but the FDA has no right to disclose that fact (i.e. the action of a 'hold' on an IND) to the public!
Good point! EU although said ARIA can continue to market but place heavy restriction with a restricted label on the product!!!! BEWARE!!!!
So Ataluren study with more patients is a junk but 10 patient SRPT study is not????
SCAM is uncovered by the FDA! So SRPT enrolled 10 health DMD patients in their exploratory study and tried to fool the FDA, DMD patient groups and the public!....Time is critical, go tell SRPT to start that p3 trial ASAP!!! and prove that this drug is not a scam....
Don't go wasting the senator time got much more important things to do than to address your concerns about losing money betting on scam!
Instead demand SRPT as to why they are delaying to start the p3 trial to prove that this drug is not a scam! 10 patient exploratory study with healthy DMD patients will not cut it!!!
Don't get your hope too high! FDA cannot release or disclose any information that a company submits to the FDA. On the contrary, it is left to the company whether or not admit whether they submitted the info or not submitted. One thing from the regulatory perspective, a clinical study report with all the details will have to be submitted to the FDA within one year of completion of the study. Also, every year a company is obligated to submit details of the current status of the study with details of the data. So FDA has the data but they cannot admit that they have the data and seen it until RNA publicly discloses. This is per US law enacted by the Congress way back!! So shut the pie hole with all your conspiracy theories......SRPT is dead with their gambled scam, and just live with it! the gamble is over and its time to start your p3 study and prove that SRPT is real not a SCAM!
The history speaks otherwise!
FDA reviews are world renowned Vs Europe who relies on so called experts which led to Thalidomide disaster and most recently Accomplia embarrassment.....
Even FDA allows Arias drug, it will be severely restricted marketing/sales..........label changes with REMS is definite possibility if the FDA allows this drug back in....
Yoh, Pazdur originally came from MD Anderson....so much you know!
Why kill cancer patients faster with fatal toxicities???
Thalidomide...premature approval later to be yanked
Most recently, Accomplia from sanofi, approved first but later yanked after 8 months in market. FDA never approved the above drugs and saved American citizens from deaths/deformities.
People already suffering from deadly cancer why subject them to faster deaths from fatal toxicities?
The following are facts:
1) Greater than 90% small/emerging biotech companies are failure
2) Companies staffed with fundamental scientists (PhD/MDs in lab experiments or in vitro/in vivo science stage) at the management level is where most of the failure occur
3) People who claim that they worked on some approved therapies in their Bios, please pay more attention and you will find that he/she is far from the actual drug development staff who really enabled the approval.
4)Drug development/Regulatory development is only gained via actual experience and no school out there teaches these regulatory and scientific standards that pushes drug from IND enabling studies transitioning into Phase 1 to Phase 2 or Phase 3 and navigating through FDA/EMA regulatory authorities.
5) Some of theses inexperienced scientists and I have seen a nobel laureates canot differentiate what an API Vs Drug product or what is a prodrug Vs Identical new molecular entities from the regulatory development science perspectives causing immense delays as well as premature failures or sometimes spending too much money on false positive signals. (AVEO, Delcath, Aria, SRPT ....etc)
So my bottom line analysis says that Epizyme is very risky stock because none of the management members has actually saw a drug transition from early development all the way to approval. PhDs/MDs are mostly academic experienced or fundamental science experience in industry who are only capable to look good on paper and perhaps good in publishing papers but certainly not drug development experts. I will not invest in this stock in its present form.