The US Food and Drug Administration (FDA) has awarded 4D Molecular Therapeutics (4DMT) a rare paediatric designation for the biopharma’s cystic fibrosis (CF) gene therapy candidate 4D-710.

As per the designation, 4DMT will be eligible to receive a priority review voucher if 4D-710 is approved by the FDA.

Recipients can redeem priority review vouchers themselves for any chosen drug or can instead sell them on to other pharma companies. Slashing four months off FDA review time, vouchers currently sell for about $100m.

4DMT’s candidate is being evaluated in the Phase I/II AEROW trial in patients with CF. CF is a lung-damaging disease that affects roughly 40,000 people in the US alone. An update of interim data from the trial is expected in mid-2024, based on a 23 January press release.

The gene therapy has already produced early positive results improving quality-of-life and spirometry-measured outcomes.

4D-710 is intended for CF patients who either are not eligible for or cannot tolerate currently approved CF transmembrane conductance regulator (CFTR) modulators. There are about 2,000 known mutations in the CFTR gene.

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4DMT’s co-founder and CEO David Kirn said: “[The designation] 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.”

Vertex Pharmaceuticals currently dominates the CF treatment space with four approved FDA-approved CFTR modulators, including Trikafta (elexacaftor/ivacaftor/tezacaftor) and Kalydeco (ivacaftor). It also has Symdeko (tezacaftor/ivacaftor) and Orkambi (umacaftor/ivacaftor). Vertex expects full-year 2023 sales from its CF treatments to be between $9.7bn and $9.8bn.

4DMT says its therapy has the potential to treat a broad range of people with CF, independent of the specific CFTR mutation. Comprised of the company’s A101 vector and CFTR gene with a partially deleted R domain, the candidate is delivered via aerosol to ensure expression within the lung airway epithelial cells.  The drug has demonstrated CFTR expression significantly above normal in treated patients, according to the 23 January press release.

4DMT is also open to developing the drug in combination with CFTR modulators, citing that lung function improvement in patients only taking CFTR modulators is incomplete.

An update on pivotal trial planning for the candidate is expected in Q1 2024.

Kirn added: “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.”

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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