FDA response to Kmail (YMB) and biopearl’s related post

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huntingonthebluffs
Posts: 246
Joined: Wed Feb 24, 2016 12:00 am

FDA response to Kmail (YMB) and biopearl’s related post

Post by huntingonthebluffs » Wed Jan 16, 2019 11:15 pm

While redundant for those of us who follow some of the GERN YMB posts, I felt these two posts were of great interest to all those following the progress and activities around efforts to commercialize Imetelstat. There were also several interesting and useful replys to both post and if you access the YMB for GERN you may want to review them as well.

In addition, I think it points out the types of efforts that retail investors and other stakeholders can take to begin to move the needle. Given the number of us and our collectively abilities there is much more we can do in supportive roles. I am hopeful that the ImetelChat thread that karagozoglu12345 has opened for “Brainstorming” will be effective at gathering more input and ideas on what subjects and avenues to pursue.

Kmall’s post
Kmail's response this morning from the FDA concerning Imetelstat..........

CDER DRUG INFO
7:49 AM (8 minutes ago)
to me

Dear Kmall,

Thank you for writing to Commissioner Dr. Scott Gottlieb at the Food and Drug Administration (FDA). This is in response to your email dated December 11, 2018 requesting that FDA fast track imetelstat for compassionate use. Your email was forwarded to the Division of Drug Information in FDA’s Center for Drug Evaluation and Research (CDER) for a response.

Please accept our sincere condolences for the loss of your best friend, Matt Sapolin, several years ago.

We take concerns about drug treatments for cancer very seriously. The FDA’s mission is to assure that safe and effective drugs are made available to the American people as quickly as possible. The FDA is committed to providing timely access to potentially useful medical treatments for seriously ill patients, as well as to working for speedy approval of new drug products while maintaining high, scientifically based, safety and efficacy standards.

Please understand that information about any investigational drug or an application under review is considered confidential and can only be disclosed to (or by) the drug sponsor as per regulations 21 CFR part 314.430 and 21 CFR part 312.130, respectively. These citations are Federal regulations that do not allow us to release information for any product that is not approved by FDA. You can read these regulations online at www.ecfr.gov.

We note that Geron Corporation has issued press releases about imetelstat. (Please note the FDA does not endorse information found on non-government websites.)

While we cannot disclose information about imetelstat, we can provide general information regarding Fast Track, clinical trials and patient access to investigational drugs.

Fast Track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose of Fast Track is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. A Fast Track designation for an investigational drug must be requested by the drug company and the request can be initiated at any time during the drug development process. FDA will review the request and make a decision within sixty days based on whether the drug fills an unmet medical need in a serious condition.

The primary way for a patient to receive a potentially promising but unapproved (investigational) drug is as a participant in a controlled clinical trial. Participating in a clinical trial helps to demonstrate a drug’s safety and effectiveness and ensures the foundation for the drug’s future use in patients. Clinical trial information is published online by the US National Library of Medicine at https://www.clinicaltrials.gov/. Patients and their physicians can use this web site to see if there are clinical trials that are recruiting new patients, and to find out if a patient may be eligible to enroll. The list of trials involving imetelstat indicates that a phase 3 trial involving imetelstat is currently recruiting.

When patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access. See Expanded Access (Compassionate Use) on our website for additional information and procedures. Note that the company developing the investigational drug makes an expanded access policy and determines whether to make an investigational drug available through expanded access. The 21st Century Cures Act requires that a company developing investigational drugs (including biologics) shall make its policy regarding evaluating and responding to requests for expanded access public and readily available. Posting this information on the Reagan-Udall Foundation’s Expanded Access Navigator, including links to a company’s own web page describing its policy for the investigational drug, can be used to satisfy this requirement.

On May 30, 2018, President Trump signed into law the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 (Right to Try Act). This new law amends the Federal Food, Drug, and Cosmetic Act to establish an alternative pathway to FDA’s expanded access program for patients to access certain unapproved products. This law is aimed at increasing access to unapproved, investigational drugs and biological products for patients diagnosed with life-threatening diseases or conditions who have exhausted approved treatment options and who are unable to participate in a clinical trial involving the investigational product, as certified by a physician. For more information, please see our Right to Try web page.
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ReplyReplies (20)

Andrew's post:
Kmall, very much appreciate your efforts. I believe they are not in vain and we all have had personal losses related to cancer. Anything we can do to raise awareness in the general public and specifically within the FDA has value. The FDA is a bureaucracy.
It is not impossible that Janssen met with them before making the CD and got guidance that spleen end point was the gold standard only to find two months later that another overarching power in the FDA would come out with guidelines highlighting the importance of OS. Maybe they didn't hear Dr. Mancarenhas say that 10% spleen reduction had important clinical value. Maybe because he couldn't say it until ASH. We keep hearing about orphan designation, fast track, new FDA, expedited approvals. If not Imetelstat then what? Unfortunately we have an FDA firing on one cylinder thanks to the shutdown. Quick back of the envelope would show that more than a hundred thousand (!) blood transfusions could easily be saved (conservative estimate) a year with approval. Disease modification, hmmm what could that mean? Longer life? Less AML? Not to mention combination therapy. Warren Buffet said one should look for companies so good that even and idiot could run them because sooner or later and idiot will run them. I reference Janssen in this case not Geron. Scarlett is plenty smart and he knows what he has. So do we, all one needs to do is look at the presentations at ASH and EHA. Not just those from Geron/Janssen but anything else pertaining to the MF/MDS space. Ask yourself if you saw anything close: I saw nothing that added life to MF patients and I saw nothing that reduced transfusion burden in patients with high transfusion burdens. I did see a plenary session for the Medalist trial that had a bar so low that 13% of the placebo patients achieved TI all by themselves. Kmall, necmi and others keep doing what you are doing. Best Regards, bp (one of the andrew's)
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