Protara Therapeutics Receives Both FDA Breakthrough Therapy and Fast Track Designations for TARA-002 in Pediatric Patients with Lymphatic Malformations

Rhea-AI Summary

Protara Therapeutics (NASDAQ: TARA) announced that the U.S. FDA granted both Breakthrough Therapy and Fast Track designations for TARA-002 to treat pediatric macrocystic and mixed cystic lymphatic malformations (LMs) on January 5, 2026.

The FDA also selected TARA-002 for the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program, and the program will cover manufacturing for both the company’s LMs and non-muscle invasive bladder cancer programs. TARA-002 previously received Rare Pediatric Disease designation. Management expects to meet with the FDA to define a path to registration for TARA-002 in LMs in the first half of 2026.

Positive

• Breakthrough Therapy designation granted by FDA
• Fast Track designation granted by FDA
• Selected for FDA CDRP manufacturing pilot
• Previously held Rare Pediatric Disease designation

Negative

• No clinical efficacy or safety readouts disclosed in the announcement
• Regulatory path to registration remains to be defined with FDA in H1 2026

Market Reality Check

$5.75 Last Close

Volume Volume 416,925 is 0.4x the 20-day average of 1,048,952, indicating subdued trading ahead of the news. low

Technical Shares at $5.38 are trading above the 200-day MA of $4.12 and sit 31.2% below the 52-week high of $7.82.

Peers on Argus 1 Up 1 Down

Select biotech peers showed mixed moves, with EQ down ~5.23% and IOBT up ~4.31%. No clear synchronized sector trend appears around this TARA headline.

Historical Context

Date	Event	Sentiment	Move	Catalyst
Dec 08	Equity offering close	Negative	+0.3%	Closing of $75M underwritten public offering and related dilution.
Dec 04	Equity offering pricing	Negative	+0.7%	Pricing of $75M public equity offering at $5.75 per share.
Dec 04	Equity offering proposed	Negative	+0.7%	Announcement of proposed $75M equity raise under existing shelf.
Dec 03	Clinical data update	Positive	+9.3%	Updated Phase 2 ADVANCED-2 NMIBC data showing strong CR rates and safety.
Dec 01	Conference/webcast notice	Neutral	-8.7%	Scheduling of call and SUO presentation for new ADVANCED-2 interim data.

Pattern Detected

Recent history shows positive price alignment with strong clinical data, but equity offerings and neutral conference updates have often seen muted or divergent reactions.

Recent Company History

Over the past months, Protara has combined clinical progress in TARA-002 with significant financing activity. The Phase 2 ADVANCED-2 update on Dec 3, 2025 in NMIBC produced a strong +9.32% reaction, contrasting with largely flat responses to the December $75 million equity offering sequence. An earlier conference-call announcement on Dec 1, 2025 drew a negative move. Today’s new FDA designations for TARA-002 in pediatric LMs build directly on this pattern of clinically oriented catalysts.

Market Pulse Summary

This announcement grants TARA-002 both FDA Breakthrough Therapy and Fast Track designations in pediatric LMs and accepts it into a CMC readiness pilot, highlighting regulatory recognition of unmet need and clinical promise. It complements prior positive NMIBC data and recent financings aimed at advancing TARA-002. Investors may monitor upcoming FDA interactions on the registration path, evolution of trial data in LMs and NMIBC, and future capital-raising activity.

Key Terms

breakthrough therapy designation regulatory

"has granted both Breakthrough Therapy and Fast Track designations for TARA-002"

A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.

fast track regulatory

"has granted both Breakthrough Therapy and Fast Track designations for TARA-002"

A fast track designation is a regulatory label that speeds up the review and communication between a drug developer and regulators for treatments addressing serious illnesses or unmet medical needs. For investors, it matters because it can shorten development time and reduce regulatory delays—like getting a VIP lane at the airport—raising the chance of earlier market access and potential revenue, though it does not guarantee approval.

lymphatic malformations medical

"for the treatment of pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs)"

Lymphatic malformations are abnormal clusters or sacs formed when the body’s lymphatic “plumbing” (the vessels that drain fluid and fight infection) grows or connects incorrectly, often causing swelling, pain, infection risk or breathing and feeding problems. Investors watch them because they create demand for specialized treatments, procedures and long-term care, influence clinical trial designs and regulatory reviews, and can affect reimbursement and market potential for drugs, devices or therapies targeting these conditions.

chemistry, manufacturing, and controls regulatory

"the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program"

Chemistry, manufacturing, and controls (CMC) is the detailed documentation of how a drug or medical product is made, tested, and kept consistent — like a recipe, factory checklist, and quality-control plan combined. Investors care because strong CMC means regulators are more likely to approve the product and the company can reliably scale production, while weak or incomplete CMC raises the risk of approval delays, production problems, extra costs, or recalls.

non-muscle invasive bladder cancer medical

"manufacturing for both the company’s LMs and non-muscle invasive bladder cancer (NMIBC) programs"

A form of bladder cancer that is confined to the inner lining of the bladder and has not grown into the deeper muscle layer; think of it like a stain on wallpaper rather than damage to the wall’s studs. It matters to investors because it has different treatment, monitoring and recurrence patterns than deeper cancers, driving demand for repeated outpatient procedures, local therapies and diagnostic tests that affect revenue, trial design and pricing dynamics in healthcare markets.

cell-based therapy medical

"TARA-002, the Company’s investigational cell-based therapy, for the treatment of pediatric patients"

Cell-based therapy uses living cells as the active treatment, delivering them into a patient to replace, repair or instruct damaged tissue or immune responses—think of sending a living repair crew into the body. For investors this matters because these therapies can offer high-value, potentially long-lasting medicines but also come with unique risks and costs tied to complex manufacturing, strict regulation, clinical trial success, and scalability.

AI-generated analysis. Not financial advice.

TARA-002 selected for FDA manufacturing development and readiness pilot program

NEW YORK, Jan. 05, 2026 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (Nasdaq: TARA), a clinical-stage company developing transformative therapies for the treatment of cancer and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy and Fast Track designations for TARA-002, the Company’s investigational cell-based therapy, for the treatment of pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs). In addition, the FDA has selected TARA-002 to participate in the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program, which aims to support CMC development of products with expedited clinical development timeframes and provide patients with earlier access.

“Receiving these important FDA designations and invitation to participate in the CDRP program highlights the significant unmet need among pediatric patients with LMs and underscores our belief that TARA-002 could serve as a meaningful treatment option for this underserved patient population,” said Jesse Shefferman, Chief Executive Officer of Protara Therapeutics. “We look forward to continuing to work with the Agency to bring this promising therapy to patients as expeditiously as possible and expect to meet with the FDA to define the path to registration for TARA-002 in LMs in the first half of this year.”

The FDA’s Breakthrough Therapy designation is a process designed to expedite the development and regulatory review of drugs or biologics that are intended to treat serious conditions where preliminary clinical evidence indicates that the drug or biologic may demonstrate substantial improvement on at least one clinically significant endpoint over available therapy. The FDA’s Fast Track program is intended to facilitate the development and expedite the review of new drugs and biologics designed to treat serious conditions with unmet medical needs.

TARA-002 was previously granted Rare Pediatric Disease designation for the treatment of LMs.

The FDA created the CDRP Program to facilitate CMC development for therapies with compressed clinical development timeframes based on the anticipated clinical benefits of earlier patient access to the therapy. The initiative is designed to promote earlier and more structured engagement between sponsors and the FDA on CMC development strategies, and since its inception, has led to increased collaboration with the FDA so that sponsors can confidently scale up manufacturing capacity while clinical development is ongoing. TARA-002 has been accepted into this program, which will involve manufacturing for both the company’s LMs and non-muscle invasive bladder cancer (NMIBC) programs.

About TARA-002 in LMs

TARA-002 is an investigational, genetically distinct strain of streptococcus pyogenes that is inactivated while retaining its immune-stimulating properties. It was developed from the same master cell bank as OK-432, which was originally granted marketing approval by the Japanese Ministry of Health for the treatment of LMs and has been the standard of care in Japan for 30 years. In addition, OK-432 was studied in a large Phase 2 trial in LMs in over 500 patients with significant clinical success. TARA-002 has been granted Rare Pediatric Disease, Breakthrough Therapy and Fast Track designations by the U.S. Food and Drug Administration for the treatment of LMs.

About Lymphatic Malformations

LMs are rare, congenital malformations of lymphatic vessels resulting in the failure of these structures to connect or drain into the venous system. Most LMs are present in the head and neck region and are diagnosed in early childhood during the period of active lymphatic growth, with more than 50% detected at birth and 90% diagnosed before the age of three years. The most common morbidities and serious manifestations of the disease include compression of the upper aerodigestive tract, including airway obstruction requiring intubation and possible tracheostomy dependence; intralesional bleeding; impingement on critical structures, including nerves, vessels and lymphatics; recurrent infection; and cosmetic and other functional disabilities.

About Protara Therapeutics, Inc.

Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin, as well as a Phase2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials (including the timing of any particular phases of such trials and the timing of the announcement of any data produced during such trials or phases thereof); statements related to expectations regarding interactions with the U.S. Food and Drug Administration (FDA); Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year and future periods. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara's filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management's assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law.

Company Contact:

Justine O'Malley
Protara Therapeutics
Justine.OMalley@protaratx.com
646-817-2836

FAQ

What FDA designations did Protara (TARA) receive for TARA-002 on January 5, 2026?

Protara received both Breakthrough Therapy and Fast Track designations from the FDA for TARA-002 in pediatric LMs.

What does FDA selection for the CDRP program mean for Protara (TARA) manufacturing timelines?

Selection for the CDRP program means FDA will engage on CMC development and manufacturing readiness for TARA-002 to support expedited clinical timelines.

Will TARA-002's CDRP enrollment cover other Protara (TARA) programs?

Yes; the CDRP participation will involve manufacturing for both the LMs program and the NMIBC program.

When does Protara (TARA) expect to meet the FDA to define the registration path for TARA-002?

The company expects to meet with the FDA to define the path to registration for TARA-002 in the first half of 2026.

Has Protara (TARA) already received Rare Pediatric Disease designation for TARA-002?

Yes; TARA-002 was previously granted Rare Pediatric Disease designation for the treatment of lymphatic malformations.