PIERRE FABRE PHARMACEUTICALS STATEMENT REGARDING RECEIPT OF COMPLETE RESPONSE LETTER FOR TABELECLEUCEL BIOLOGICS LICENSE APPLICATION FROM THE U.S. FOOD AND DRUG ADMINISTRATION

Rhea-AI Summary

Pierre Fabre Pharmaceuticals (collaborating on ATRA program) announced receipt of a Complete Response Letter (CRL) from the U.S. FDA on Jan 9, 2026, stating the tabelecleucel Biologics License Application cannot be approved in its present form.

The company says the FDA acknowledged resolution of a prior GMP deficiency but now requests a new study, reversing prior acceptance of the single‑arm ALLELE study and creating an unexpected change after multi‑year dialogue. Pierre Fabre intends to engage the FDA with Atara Biotherapeutics and continue expanded access while pursuing a path toward accelerated approval.

Positive

• FDA acknowledged the prior GMP deficiency had been resolved
• Resubmission was accepted in July 2025 and given accelerated approval status
• Ongoing Expanded Access Program continues to provide patient access
• Approval and real‑world use exist in multiple countries outside the US

Negative

• FDA now requests a new study instead of relying on the ALLELE study
• CRL prevents current BLA approval and delays U.S. market access
• Agency's change may hinder evidence generation for ultra‑rare populations

News Market Reaction

-56.99% 5.8x vol

81 alerts

-56.99% News Effect

+3.1% Peak Tracked

-71.0% Trough Tracked

-$131M Valuation Impact

$99M Market Cap

5.8x Rel. Volume

On the day this news was published, ATRA declined 56.99%, reflecting a significant negative market reaction. Argus tracked a peak move of +3.1% during that session. Argus tracked a trough of -71.0% from its starting point during tracking. Our momentum scanner triggered 81 alerts that day, indicating high trading interest and price volatility. This price movement removed approximately $131M from the company's valuation, bringing the market cap to $99M at that time. Trading volume was exceptionally heavy at 5.8x the daily average, suggesting significant selling pressure.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

CRL receipt date: January 9, 2026 Prior CRL date: January 15, 2025 Dialogue duration: more than five years

3 metrics

CRL receipt date January 9, 2026 Date FDA issued Complete Response Letter for tabelecleucel BLA

Prior CRL date January 15, 2025 Date of earlier CRL citing single GMP-related deficiency

Dialogue duration more than five years Period of discussions with FDA regarding tabelecleucel program

Market Reality Check

Price: $4.54 Vol: Volume 843,003 is 5.47x t...

high vol

$4.54 Last Close

Volume Volume 843,003 is 5.47x the 20-day average of 154,243, indicating elevated trading activity ahead of/around this CRL news. high

Technical Shares at $13.67 are trading above the 200-day MA of $11.33, despite a 21.62% single-day decline.

Peers on Argus

ATRA fell 21.62% while key biotech peers showed mixed, mostly modest moves: CSBR...

1 Up

ATRA fell 21.62% while key biotech peers showed mixed, mostly modest moves: CSBR +0.30%, IMUX +0.44%, OVID -4.02%, QNCX -2.18%, STTK +1.71%. With no peers down comparably, the move appears stock-specific to the tabelecleucel CRL.

Historical Context

5 past events · Latest: Nov 12 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Nov 12	Earnings & BLA update	Positive	-0.7%	Q3 results plus FDA Priority Review and PDUFA date for tabelecleucel.
Nov 03	BLA transfer news	Positive	-6.7%	Pierre Fabre assumes BLA and global responsibilities for tabelecleucel.
Sep 03	Board changes	Neutral	+1.4%	Board reshaping as company focuses on Ebvallo milestones and CAR T assets.
Aug 11	Earnings & Priority Review	Positive	+11.8%	Q2 profitability, Priority Review, and expected <b>$40M</b> approval milestone.
Jul 24	FDA acceptance	Positive	-0.2%	FDA acceptance and Priority Review of tabelecleucel BLA with PDUFA date.

Pattern Detected

Recent history shows repeated negative or muted reactions even to seemingly positive regulatory milestones, suggesting sensitivity around tabelecleucel’s U.S. path.

Recent Company History

Over the past six months, ATRA’s story centered on tabelecleucel and its U.S. regulatory trajectory. FDA acceptance and Priority Review with a January 10, 2026 PDUFA date and the transfer of BLA responsibilities to Pierre Fabre were highlighted in multiple releases, alongside expectations for a $40M milestone upon approval. Despite these milestones, several announcements drew flat or negative reactions. Today’s Complete Response Letter overturns prior momentum around the BLA and directly challenges those earlier expectations.

Market Pulse Summary

The stock dropped -57.0% in the session following this news. A negative reaction despite prior optim...

Analysis

The stock dropped -57.0% in the session following this news. A negative reaction despite prior optimism about tabelecleucel fits the historical pattern where several seemingly positive regulatory milestones were followed by flat or negative moves. The new Complete Response Letter removed the near-term pathway that underpinned expectations for a $40M approval milestone and earlier Priority Review momentum. Given past sensitivity to tabelecleucel news and a concentrated pipeline story, a sharp decline after this setback reflected reassessment of the program’s U.S. outlook.

Key Terms

complete response letter, biologics license application, u.s. food and drug administration, gmp, +4 more

8 terms

complete response letter regulatory

"received a Complete Response Letter (CRL) from the U.S. Food and Drug"

A complete response letter is an official communication from a drug or medical-device regulator, such as the U.S. Food and Drug Administration (FDA), telling a company that a marketing application cannot be approved in its current form and listing the specific deficiencies to be fixed. For investors it matters because it pauses or delays a product’s path to market—like a building inspector issuing a list of repairs before a certificate of occupancy—affecting revenue timing, costs and stock value.

biologics license application regulatory

"unable to approve the tabelecleucel Biologics License Application (BLA) in its"

A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.

u.s. food and drug administration regulatory

"received a Complete Response Letter (CRL) from the U.S. Food and Drug"

The U.S. Food and Drug Administration is the federal agency that evaluates and enforces safety, effectiveness and labeling standards for medicines, medical devices, vaccines, food and related products before they reach consumers. For investors it matters because FDA approvals, warnings or recalls determine whether a product can be sold, how quickly it reaches the market and how costly compliance will be—changes that directly affect a company’s revenue, costs and stock value.

gmp technical

"identified a single GMP-related deficiency and raised no concerns"

Good Manufacturing Practice (GMP) is a set of regulatory standards and procedures that ensure products—especially medicines, medical devices, and related goods—are consistently made to meet safety, quality, and purity requirements. For investors, GMP compliance is like a factory’s hygiene and checklist system: it reduces the risk of product recalls, regulatory fines, and production stoppages, supports market access, and signals more reliable, lower-risk operations that can protect revenue and reputation.

accelerated approval regulatory

"the FDA granted tabelecleucel accelerated approval status."

Accelerated approval is a process that allows new medical treatments to be approved more quickly than usual if they address serious or life-threatening conditions and show promising early results. For investors, it signals that a treatment may reach the market sooner, potentially boosting a company's prospects, but it also involves some uncertainty since full evidence of effectiveness is still being gathered.

single-arm medical

"no longer considers the previously accepted single-arm ALLELE study to be"

A single-arm study is a clinical trial that gives all participants the same treatment and does not include a separate comparison group or placebo. Think of it like testing a new recipe by serving it to diners without offering a control dish — you can see how people respond, but you can’t directly compare results to another option. For investors, single-arm trials can speed development and reduce cost but leave more uncertainty about how a treatment stacks up against existing therapies and how regulators will view the evidence.

expanded access program regulatory

"committed to making tabelecleucel available to patients through our Expanded Access Program."

A program that allows patients with serious or life‑threatening conditions to receive an experimental drug or therapy before it is fully approved by regulators, when they cannot join clinical trials. Investors care because expanded access can change a treatment’s market perception, create early real‑world safety or demand signals, and affect regulatory timelines and potential revenue — like a pre‑order system that also reveals how the product performs outside controlled testing.

epstein-barr virus–positive post-transplant lymphoproliferative disease medical

"patients with Epstein-Barr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD)"

A serious complication that can occur after an organ or stem-cell transplant when immune-suppressing drugs let Epstein-Barr virus (EBV) trigger uncontrolled growth of a patient’s white blood cells, forming tumors or masses. Investors care because its occurrence affects demand for, and regulatory review of, drugs and diagnostics, can change clinical trial results and commercial prospects, and may create liabilities or market opportunities—like weeds suddenly spreading in a garden when the fence is down.

AI-generated analysis. Not financial advice.

SECAUCUS, N.J., Jan. 12, 2026 /PRNewswire/ -- On January 9, 2026, Pierre Fabre Pharmaceuticals, Inc. received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) stating that the Agency is unable to approve the tabelecleucel Biologics License Application (BLA) in its present form.

We are surprised and deeply disappointed by the FDA's decision, particularly given the urgent and life-threatening unmet medical need faced by patients with Epstein-Barr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD) after failure of standard-of-care therapy. These patients have no FDA-approved treatment options and a life expectancy often measured in weeks to months.

The BLA was resubmitted following clear alignment with the FDA on the acceptability of the resubmission criteria and fulfillment of the conditions outlined in the January 15, 2025, CRL, which identified a single GMP-related deficiency and raised no concerns regarding safety, efficacy, or trial design. Upon acceptance of the resubmission in July 2025, the FDA granted tabelecleucel  accelerated approval status.

In the new CRL, despite acknowledging that the GMP issue had been resolved and raising no safety concerns, the FDA stated that it no longer considers the previously accepted single-arm ALLELE study to be adequate to support accelerated approval and requested a new study. This represents a significant and unexpected change in position, and one that is contrary to extensive dialogue with the Agency over more than five years. 

We are concerned that this decision may have far-reaching consequences for the development of rare disease treatments, effectively creating barriers for generating clinical evidence within a unique patient population with ultra-rare conditions thereby significantly delaying—or preventing altogether— patient access to urgently needed therapies.

We firmly believe that tabelecleucel represents an important treatment advance for patients with EBV+ PTLD and that the totality of data supports its efficacy and safety. We intend to engage with the FDA to urgently pursue a path forward, in collaboration with Atara Biotherapeutics (Nasdaq: ATRA) and our clinical and patient partners, to enable timely accelerated approval of tabelecleucel.  We continue to be committed to making tabelecleucel available to patients through our Expanded Access Program.

Approval and real-world use of tabelecleucel over several years in multiple countries outside the United States further support its clinical value. We remain fully committed to securing approval of this critical treatment option for U.S. patients and the physicians who care for them.

About Pierre Fabre Pharmaceuticals and Pierre Fabre Laboratories

The mission of Pierre Fabre Pharmaceuticals (PFP) is to deliver breakthrough therapies in oncology and rare diseases to patient populations with high unmet needs and limited treatment options. Our belief is that every time we care for a single person, we make the whole world better.

PFP is the US pharmaceutical subsidiary of Pierre Fabre Laboratories, a foundation-owned company with seven decades of impact. Pierre Fabre Laboratories is a global healthcare company, established in 43 countries, over 10,000 employees, and with products distributed in 120 territories across the globe.

The Pierre Fabre Laboratories foundation ownership enhances the ability of the company to create long-term value for patients. Partnerships and acquisitions drive its innovative precision treatment pipeline and are enabled by the unique corporate structure.

Building on the legacy of Pierre Fabre Laboratories, innovation is the life blood of PFP and patient experience© drives everything the company does. PFP aspires to design and develop therapeutic solutions inspired by patients and healthcare professionals; draw on science and nature as perpetual sources of inspiration; develop long-term partnerships with researchers and innovators worldwide; and place pharmaceutical ethics and climate transition at the heart of our action.

Pierre Fabre Pharmaceuticals has therapies in development for Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD), NRAS-mutant melanoma, non-small cell lung cancer with mutation or amplification of MET, and X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED). Pierre Fabre Pharmaceuticals is headquartered in Secaucus, NJ.

For more information, visit www.PierreFabrePharmaceuticals.com, www.Pierre-Fabre.com, @Pierre Fabre Oncology.

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SOURCE Pierre Fabre Pharmaceuticals

FAQ

What did Pierre Fabre announce about the tabelecleucel BLA for ATRA on Jan 9, 2026?

The company received an FDA Complete Response Letter saying the BLA cannot be approved in its present form.

Why did the FDA issue the CRL for tabelecleucel (ATRA) despite GMP resolution?

The FDA acknowledged GMP resolution but stated the single‑arm ALLELE study is no longer adequate and requested a new study.

How will the CRL affect patient access to tabelecleucel in the U.S. for ATRA?

The CRL prevents current approval and may delay or restrict timely U.S. access pending additional study or regulatory engagement.

What are Pierre Fabre and Atara Biotherapeutics planning after the Jan 9, 2026 CRL for ATRA?

They intend to urgently engage the FDA, work with clinical and patient partners, and pursue a path toward accelerated approval.

Is tabelecleucel available outside the U.S. for patients related to ATRA work?

Yes; the company notes approval and real‑world use of tabelecleucel in multiple countries outside the United States.