This article provides a brief summary of the FDA new drug approvals in July 2025, highlighting the emerging stars in the pharmaceutical world that bring new hope to life and health:
Recently, PTC Therapeutics announced that the FDA has approved Sephience (sepiapterin) for the treatment of pediatric and adult patients with phenylketonuria (PKU). This approval covers a broad indication for adults and children aged 1 month and older with high phenylalanine levels (HPA) who respond to sepiapterin. The FDA approval is primarily based on significant efficacy and safety data from the Phase 3 APHENITY clinical trial, with sustained treatment effects verified in the APHENITY long-term extension study. The APHENITY trial results were published in the renowned medical journal The Lancet. Analysis showed that patients treated with sepiapterin had an average 63% reduction in phenylalanine. Over 80% of patients achieved blood phenylalanine levels below the target thresholds established by the US and EU for children and adults with PKU.
LEO Pharma recently announced FDA approval of Anzupgo (delgocitinib) cream (20 mg/g) for the topical treatment of adult patients with moderate to severe chronic hand eczema (CHE) who are unresponsive to or unsuitable for topical corticosteroids. According to the press release, Anzupgo cream is the first FDA-approved therapy specifically for moderate to severe chronic hand eczema in adults. Anzupgo is a “first-in-class” topical pan-JAK inhibitor that suppresses activation of the JAK-STAT signaling pathway involved in chronic inflammatory skin disease lesions.
Previously published Phase 3 DELTA 1 and DELTA 2 trials evaluated the safety and efficacy of Anzupgo versus placebo for CHE treatment. Treatment success was defined as achieving Investigator Global Assessment for CHE Treatment Success (IGA-CHE TS) scores of 0 (clear skin) or 1 (almost clear), with at least a two-grade improvement from baseline.
At week 16, a greater proportion of patients in the Anzupgo groups met the treatment success criteria compared to placebo. In DELTA 1, 20% of Anzupgo patients vs. 10% placebo patients achieved treatment success; in DELTA 2, 29% vs. 7% (both trials p ≤ 0.0055).
KalVista Pharmaceuticals announced on July 7 that the FDA approved Ekterly (sebetralstat, 300mg) tablets for the treatment of acute attacks of hereditary angioedema (HAE) in adults and pediatric patients aged 12 years and older. Sebetralstat is an oral plasma kallikrein inhibitor targeting the kallikrein-kinin system cascade. By inhibiting cleavage of high molecular weight kininogen, it reduces bradykinin production to relieve HAE symptoms. Notably, Ekterly is the first and only FDA-approved oral on-demand therapy for acute HAE attacks in patients aged 12 and older in the US.
The approval is based on data from the randomized, triple-crossover Phase 3 KONFIDENT trial (ClinicalTrials.gov Identifier: NCT05259917), which assessed efficacy and safety in children and adults with type 1 or type 2 HAE aged ≥12 years.
Participants were randomized to sebetralstat 300mg, sebetralstat 600mg, or placebo.
Results showed that sebetralstat 600mg significantly accelerated symptom relief compared to placebo. Median time to symptom relief in the 600mg group was 2 hours (95% CI, 1.5-2.8). The time to first significant symptom severity reduction within 12 hours post-dose (assessed by Patient Global Impression of Severity [PGI-S]) was also significantly shorter than placebo, with a median of 9.1 hours (95% CI, 3.8, not reached).
Additionally, attack resolution time (PGI-S = “none” within 24 hours post-dose) was significantly faster with sebetralstat 600mg.
Dizal Pharma announced on July 2 that the FDA granted accelerated approval to Zegfrovy (sunvozertinib) for the treatment of adult patients with locally advanced or metastatic NSCLC harboring FDA-approved detected EGFR exon 20 insertion mutations whose disease progressed during or after platinum chemotherapy.
Zegfrovy received FDA priority review and breakthrough therapy designation, and is the only approved targeted oral therapy for EGFR exon 20 insertion mutation NSCLC. This indication was granted accelerated approval based on overall response rate and duration of response. Continued approval may depend on verification and description of clinical benefit in confirmatory trials.
The FDA also approved Thermo Fisher Scientific’s Oncomine Dx Express Test as a companion diagnostic for detecting EGFR exon 20 insertion mutations in NSCLC patients.
Zegfrovy is an irreversible EGFR inhibitor developed by Dizal scientists, targeting multiple EGFR mutations with selectivity over wild-type EGFR. The approval is based on data from the WU-KONG1B trial (ClinicalTrials.gov: NCT03974022), involving 85 patients with locally advanced or metastatic EGFR exon 20 insertion mutation NSCLC who progressed during or after platinum chemotherapy. Patients received 200mg sunvozertinib orally once daily with food until disease progression or unacceptable toxicity. The confirmed overall response rate (primary endpoint) was 46% (95% CI, 35-57). Duration of response was 11.1 months (95% CI, 8.2, not estimable).
Regeneron Pharmaceuticals announced on July 2 that the FDA granted accelerated approval to Lynozyfic (linvoseltamab-gcpt) for adult patients with relapsed or refractory (R/R) multiple myeloma (MM) who have received at least four prior therapies, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Linvoseltamab is a BCMA/CD3-targeting bispecific antibody designed to link BCMA on myeloma cells with CD3 on T cells, promoting T cell activation and tumor cell killing. Developed using Regeneron’s VelocImmune technology, Lynozyfic is a fully human BCMAxCD3 bispecific antibody.
FDA approval is based on results from the pivotal Phase 1/2 LINKER-MM1 trial involving 80 patients. Independent review showed an objective response rate (ORR) of 70%, with 45% achieving complete response (CR) or better. Median time to first response was 0.95 months (range 0.5 to 6 months). Median duration of response (DoR) was not reached (95% CI: 12 months to not estimable). At a median follow-up of 13 months, the estimated 9-month DoR rate was 89% (95% CI: 77-95) and 12-month DoR rate was 72% (95% CI: 54-84).
Among patients treated at the recommended dose, 46% experienced cytokine release syndrome (CRS) (grade 3, <1%) and 54% had neurological toxicities including immune effector cell-associated neurotoxicity syndrome (ICANS) (grade 3-4, 8%). The prescribing information includes a black box warning for life-threatening CRS and neurotoxicity (including ICANS). Due to these risks, Lynozyfic is available only through a restricted program called the Lynozyfic Risk Evaluation and Mitigation Strategy (REMS).
The press release notes Lynozyfic is the first FDA-approved BCMAxCD3 bispecific antibody, dosed every two weeks starting from week 14, with dosing every four weeks if very good partial response (VGPR) or better is achieved after at least 24 weeks of treatment. The regimen includes inpatient monitoring during dose escalation (24 hours after the first and second dose escalation) to ensure safety.
