4DMT Receives Rare Pediatric Disease Designation from FDA for Aerosolized 4D-710 for Treatment of Cystic Fibrosis Lung Disease

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4D Molecular Therapeutics (Nasdaq: FDMT) announced that the U.S. FDA granted Rare Pediatric Disease Designation for aerosolized 4D-710, a treatment for cystic fibrosis lung disease. Promising clinical trial results showed significant CFTR expression levels and improved quality of life for patients. The company is also planning an interim data update and pivotal trial planning update for 2024.
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The granting of Rare Pediatric Disease Designation (RPDD) by the FDA for 4D-710, a gene therapy candidate for cystic fibrosis (CF) lung disease, is a pivotal regulatory milestone for 4D Molecular Therapeutics. RPDD can expedite the development and review process, which is crucial for conditions with unmet medical needs. The therapy's promising interim results, showing CFTR transgene expression significantly above normal levels, suggest a potential breakthrough in CF treatment. This is especially significant for patients who are ineligible for existing CFTR modulators.

From a research perspective, the data indicating durable improvement or stabilization of quality of life and pulmonary function is encouraging. However, the long-term efficacy and safety of 4D-710 must be closely monitored. The decision to explore lower doses in Cohort 3 is a strategic move to optimize the balance between efficacy and safety. The upcoming interim data update in mid-2024 will be critical in evaluating the therapy's potential and informing the design of the pivotal trial.

The RPDD for 4D-710 could have a significant financial impact on 4D Molecular Therapeutics. If the drug is approved, the company may receive a priority review voucher, which is a valuable asset that can be sold or transferred. Such vouchers have been sold for prices ranging from $67 million to $350 million in the past, providing a substantial financial incentive for the company. Investors should note the potential for increased market interest and funding opportunities as the therapy progresses.

Furthermore, the mention of dose exploration and the planned pivotal trial update in Q1 suggests that the company is actively managing its clinical development pathway. This proactive approach can enhance investor confidence, as it demonstrates a commitment to optimizing the therapeutic's profile and addressing regulatory considerations efficiently.

The development of novel treatments like 4D-710 has broader economic implications, particularly in the context of rare pediatric diseases. The costs associated with CF, both direct medical costs and indirect costs such as lost productivity, are substantial. An effective treatment can reduce these costs significantly. Moreover, advancements in gene therapy can stimulate further investment and innovation within the biotech sector, potentially leading to job creation and economic growth.

However, the high cost of developing and delivering gene therapies poses challenges regarding affordability and access. The economic analysis must consider the potential pricing strategies and reimbursement models that will be necessary to ensure that such innovative treatments are accessible to those in need, without placing an undue burden on healthcare systems.

  • 4D-710 has demonstrated promising, reproducible, CFTR expression significantly above normal levels for seven patients across Cohorts 1 & 2 (1E15-2E15 vg) and durable improvement or stabilization of quality of life & pulmonary function for three patients through 12 months in Cohort 1
  • Given above normal CFTR transgene expression (~400% of normal lung levels), dose exploration continues with evaluation of lower doses (Cohort 3 dose 5E14 vg)
  • Interim data update from Phase 1/2 AEROW clinical trial expected in mid-2024, and pivotal trial planning update expected in Q1

EMERYVILLE, Calif., Jan. 23, 2024 (GLOBE NEWSWIRE) -- 4D Molecular Therapeutics (Nasdaq: FDMT, 4DMT or the Company), a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) for the Company’s product candidate aerosolized 4D-710 for treatment of cystic fibrosis (CF) lung disease. 4D-710 is being evaluated in the Phase 1/2 AEROW clinical trial in people with CF who are not eligible for, or cannot tolerate any of, the currently approved CFTR modulators.

RPDD may be granted to investigational drugs and biologics designed to address serious or life-threatening diseases primarily affecting individuals 18 years of age and under, and meeting the definition of “rare disease or condition” (affects less than 200,000 individuals in the U.S.). If the drug or biologic is approved for marketing, 4DMT may qualify for a priority review voucher that may be transferred or sold to another sponsor.

“The Rare Pediatric Disease Designation is a very important regulatory milestone in our development path for 4D-710 and highlights the urgent need for novel therapeutic options for people living with CF lung disease, including children, especially those who are not eligible for currently available disease modifying therapies,” said David Kirn, M.D., Co-founder and Chief Executive Officer of 4DMT. “Based on the CFTR expression levels and clinical activity seen to-date, we are excited about the potential for 4D-710 to transform the lives of people with CF with an effective and durable treatment option. We continue to enroll our AEROW clinical trial and work with the CF Foundation and regulators to identify the most efficient path to advance this therapy, with preliminary feedback expected this quarter.”

About Cystic Fibrosis Lung Disease and 4D-710

Cystic fibrosis is an inherited, progressive disease caused by mutations in the CFTR gene. It affects the lungs, pancreas, and other organs. According to the CF Foundation, nearly 40,000 people in the United States and more than 105,000 people worldwide are living with cystic fibrosis, with approximately 1,000 new cases of cystic fibrosis diagnosed in the United States each year. Lung disease is the leading cause of morbidity and mortality in people with cystic fibrosis. Cystic fibrosis causes impaired lung function, inflammation and bronchiectasis and is commonly associated with persistent lung infections and repeated exacerbations due to the inability to clear thickened mucus from the lungs. People with cystic fibrosis require lifelong treatment with multiple daily medications. The complications of the disease result in progressive loss of lung function, increasing need for IV antibiotics and hospitalizations, ultimately leading to end-stage respiratory failure.

4D-710 is comprised of our targeted and evolved next generation vector, A101, and a codon-optimized CFTR∆R transgene. 4D-710 has the potential to treat a broad range of people with cystic fibrosis, independent of the specific CFTR mutation, and is designed for aerosol delivery to achieve CFTR expression within lung airway epithelial cells. 4D-710 is being initially developed for the approximately 15% of people whose disease is not amenable to existing CFTR modulator medicines (based on variant-eligibility and/or drug intolerance) targeting the CFTR protein. In people with CFTR mutations whose disease is amenable to modulator medicines, the improvement in lung function is incomplete and is variable. We therefore expect to potentially develop 4D-710 in this broader population, as a single agent and/or in combination with CFTR modulator small molecule medicines.

About 4DMT 

4DMT is a leading clinical-stage genetic medicines company focused on unlocking the full potential of genetic medicines to treat large market diseases in ophthalmology and pulmonology. 4DMT’s proprietary invention platform, Therapeutic Vector Evolution, combines the power of the Nobel Prize-winning technology, directed evolution, with approximately one billion synthetic AAV capsid-derived sequences to invent customized and evolved vectors for use in our wholly owned and partnered product candidates. Our product design, development, and manufacturing engine helps us efficiently create and advance our diverse product pipeline with the goal of revolutionizing medicine with potential curative therapies for millions of patients. Currently, 4DMT is advancing five clinical-stage and two preclinical product candidates, each tailored to address rare and large market diseases in ophthalmology, pulmonology, and cardiology. In addition, 4DMT is also advancing programs in CNS through a gene editing partnership. 4D Molecular Therapeutics™, 4DMT™, Therapeutic Vector Evolution™, and the 4DMT logo are trademarks of 4DMT.

All of our product candidates are in clinical or preclinical development and have not yet been approved for marketing by the FDA or any other regulatory authority. No representation is made as to the safety or effectiveness of our product candidates for the therapeutic uses for which they are being studied.

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Forward Looking Statements: 

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, implied and express statements regarding the therapeutic potential, and clinical benefits of 4DMT’s product candidates, as well as the plans, announcements and related timing for the clinical development of 4D-710. The words "may," “might,” "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," “expect,” "estimate," “seek,” "predict," “future,” "project," "potential," "continue," "target" and similar words or expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including risks and uncertainties that are described in greater detail in the section entitled "Risk Factors" in 4D Molecular Therapeutics’ most recent Quarterly Report on Form 10-Q as well as any subsequent filings with the Securities and Exchange Commission. In addition, any forward-looking statements represent 4D Molecular Therapeutics' views only as of today and should not be relied upon as representing its views as of any subsequent date. 4D Molecular Therapeutics explicitly disclaims any obligation to update any forward-looking statements. No representations or warranties (expressed or implied) are made about the accuracy of any such forward looking statements. 



Katherine Smith
Inizio Evoke Comms


Julian Pei
Head of Investor Relations and Corporate Communications

The ticker symbol for 4D Molecular Therapeutics is FDMT.

The designation highlights the urgent need for novel therapeutic options for people living with CF lung disease, especially children who are not eligible for currently available therapies. It may also qualify the company for a priority review voucher if the drug is approved for marketing.

The clinical trial results showed promising, reproducible CFTR expression significantly above normal levels for seven patients. Three patients also demonstrated durable improvement or stabilization of quality of life and pulmonary function through 12 months.

An interim data update from the Phase 1/2 AEROW clinical trial is expected in mid-2024, and a pivotal trial planning update is expected in Q1 2024.
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About FDMT

the mission of 4d molecular therapeutics is to design and develop transformative gene therapy products using our proprietary technology to create novel adeno-associated virus variants to be utilized as treatments for serious unmet medical conditions. our products are unlocking the full potential of gene therapy to treat, and potentially cure, genetic diseases. a large number of patients and diseases, who previously were not treatable by gene therapy, will be addressable by 4d products. 4d has one of the deepest and most diverse product pipelines in the gene therapy field. adeno-associated virus (aav) vectors have emerged as a favored delivery vehicle for gene therapy in the human body. they can deliver the genes for therapeutic proteins to accessible tissues in the body and are generally considered safe. several aav gene therapy products are in late-stage clinical development, and one product is approved in the eu (glybera, uniqure). however, these first-generation aav vectors have lim