Welcome to Scribd, the world's digital library. Read, publish, and share books and documents. See more
Download
Standard view
Full view
of .
Save to My Library
Look up keyword
Like this
2Activity
0 of .
Results for:
No results containing your search query
P. 1
2012-10-16 - REDACTED Celltex Response to FDA September 24 Letter[1]

2012-10-16 - REDACTED Celltex Response to FDA September 24 Letter[1]

Ratings: (0)|Views: 1,702|Likes:
Published by Mike Snyder

More info:

Published by: Mike Snyder on Oct 17, 2012
Copyright:Attribution Non-commercial

Availability:

Read on Scribd mobile: iPhone, iPad and Android.
download as PDF, TXT or read online from Scribd
See more
See less

10/17/2012

pdf

text

original

 
 
2401 Fountain View Dr Suite416Houston, Texas 77057T 713.590.1000F 713.780.08862401 Fountain View Dr Suite416Houston, Texas 77057T 713.590.1000F 713.780.0886
October 16, 2012
VIA EMAIL (without attachments)VIA OVERNIGHT MAIL (with attachments)
Robert A. SausvilleDirector, Office of Case ManagementRoom 1025HFM-610Rockville MD 20852Re: Response to September 24, 2012 LetterDear Mr. Sausville:Celltex Therapeutics Corporation (“Celltex”) hereby responds to FDA’s September 24,2012 letter (“September 24
th
Letter”). Celltex respects the agency’s authority, granted by thefederal Food, Drug, and Cosmetic Act (FDCA) and the Public Health Services (PHS) Act. WhileCelltex respectfully disagrees with the positions outlined in the September 24
th
Letter, Celltexbelieves that this technology’s therapeutic potential can be brought to fruition as a biologicaldrug as the agency states. Although the regulatory requirements for a biological drug are ex-cessive for an autologous mesenchymal stem cell therapy such as Celltex’s, Celltex will followthe agency’s request to treat the Mesenchymal Stem Cell (MSC) product as a biological drugand develop Investigational New Drug (IND) submissions for clinical trials.As you know, the September 24th Letter is an advisory opinion, not final agency actionon the issues it raises, inviting Celltex to explain why its product is not in violation of the FDCAand the PHS Act. Celltex would like the opportunity to explain its position, both with this letterand with its request for a meeting with FDA, as described below.
1
Celltex asks that FDA fullyand carefully evaluate all of the submissions it has made to the agency since the inspection, theinformation in this letter and the attachments, and the information presented at the requestedmeeting, as it considers this matter. Upon the completion of a fair, thorough examination of 
1
 
When FDA inspectors visited Celltex in April, and in Celltex’s communications with the agency in the five monthsthat followed, Celltex repeatedly stated its desire to maintain a cooperative relationship and an open line of com-munication with the agency. It was surprising to have the first indication of the agency’s position come in the Sep-tember 24
th
Letterletter rather than any earlier communication or conversation.
 
 
Page 2
 
that information, Celltex believes FDA will determine that Celltex’s stem cell technology canoperate in compliance as an HCT/P
2
-only product (21 CFR 1271.10).
Summary
Celltex respectfully requests a meeting with FDA for an in-depth discussion of criticalpoints raised for the first time in the September 24
th
Letter. The purpose of the meeting is togive FDA and Celltex a better understanding of both Celltex’s and the agency’s interpretation of several factors, as explained in the meeting request section below. The science and regulationsat issue here are complex, and Celltex’s operational model presents a unique structure for FDAto consider under its regulatory precedent. It would well serve both FDA and Celltex to have adialog face-to-face to achieve better understanding.Celltex has stopped enrollment and is closing out treatments in its trials for autologousstem cell therapy as described below. Celltex will contact the agency to discuss the details of that process. Celltex will also work with FDA for development of a 21 CFR 1271.10 compliantMSC HCT/P-only product in parallel with developing the product under its biological drug re-quirements and provide therapeutic MSCs in the US under IND applications.With this letter Celltex provides additional information from RNL and Human Biostar(collectively “RNL,” the contract manufacturers operating the FDA-registered Texas laboratory)concerning the April inspection results and the operations points repeated in the September24
th
Letter. That material is described below under “Inspection Observations” and in attach-ments with this submission. To address an inaccurate statement on page 8 of the September 24Letter, all of the SOPs provided in the July 31, 2012 submission and in this submission are spe-cific for RNL’s Texas laboratory operations and are under continuous review and modification asrequired. In addition, RNL has also informed us that it is making additional changes to ensurelaboratory operations are in full compliance with all applicable requirements. We include thedetails from RNL in this submission. RNL plans to have those changes completed by November15th and we will provide details within five business days of receipt of RNL’s report expected onthat date.
Confidentiality
Celltex’s operations are confidential. Its procedures, supplies, and processes are all in-tellectual property licensed from RNL (and RNL operates the Texas laboratory as the contractmanufacturer). Celltex would experience significant and irreparable injury, including financialinjury, if any of its operational and process details were made public, enabling competitors andthird parties to achieve a significant market advantage over Celltex. Thus all of the information
2
 
“Human cell, tissue, cellular or tissue-based product” (HCT/P) under 21 CFR Part 1271. 21 CFR 1271.10 defines an“HCT/P-only” category where agency pre-approval is not required for therapeutic use of a cell product when cer-tain criteria are satisfied.
 
 
Page 3
 
provided during the inspection, the documents that were given to the investigators, and all cor-respondence with FDA since the inspection, including this unredacted letter (with all attach-ments), are confidential business records exempt from public disclosure under 5 USC 552(b)(4).Please limit access to those confidential business records to only those FDA personnelwho must have access to this body of information to perform their duties. Consistent with 5USC 552(b)(4), FDA must contact Celltex prior to any public disclosure of the confidential busi-ness records, informing it of the details of the proposed disclosure.Celltex is providing a redacted version of this letter (
Exhibit A
). It requests that FDA postthe redacted version of this letter without any attachments as Celltex’s response to the Sep-tember 24 Letter on FDA’s website.
Request for a Meeting
Celltex respectfully requests a meeting pursuant to 21 CFR 10.65(c) to discuss the fol-lowing issues identified in and questions arising from the September 24
th
Letter:
x
 
FDA has identified the adipose tissue from autologous donors as the “humancell, tissue, cellular or tissue-based product” (HCT/P) at issue, not the constituentmesenchymal stem cells (MSCs);
o
 
Why is the adipose tissue being treated differently by FDA than other re-covered tissue from which HCT/Ps are isolated in compliance with1271.10, such as umbilical cord blood or semen?
o
 
If MSCs were removed from other sources such as bone marrow or pe-ripheral blood, how would that affect the application of 1271.10? Wouldthose MSCs become the HCT/P against which 1271.10 is applied?
3
 
x
 
Celltex’s April 27
th
Letter (resubmitted here as
Exhibit B
) showed the original,relevant fundamental characteristics of the MSCs are not altered (under 21 CFR1271.3(f)(2)) in any of the steps used by RNL;
o
 
Does FDA agree that the relevant characteristics of MSCs are identified inCelltex’s April 27
th
Letter (morphology, immune-phenotype, differentia-tion capability, and genetic stability)? If not, what other characteristicsnecessary to define MSCs are relevant?
o
 
Does FDA agree that the techniques described in the April 27
th
Letter fortesting those characteristics are the appropriate tests for those charac-teristics (see page 6-7 of April 27 Letter)? If FDA prefers other tests,
3
As explained below, Celltex could change the recovered tissue from which MSCs are isolated. Adipose tissue isused because it is a relatively convenient procedure for the individual and offers a plentiful supply of MSCs relativeto the volume of tissue removed compared to other sources of MSCs.

You're Reading a Free Preview

Download
/*********** DO NOT ALTER ANYTHING BELOW THIS LINE ! ************/ var s_code=s.t();if(s_code)document.write(s_code)//-->