FDA News Recap: Novel Drug Approvals in First Half of 2025

Published on: July 2, 2025

A review of the 16 novel drugs approved by the US Food and Drug Administration during the first half of 2025, with links to coverage from MJH Life Sciences publications.

The first half of 2025 brought a wave of innovation across the medical community, with 16 novel drug approvals signaling major strides in disease treatment and management across a wide range of specialties.

From the long-awaited arrival of new targeted therapies in breast and lung cancer to groundbreaking advances in rare diseases like hereditary angioedema and neurofibromatosis type 1, this wave of approvals underscores the growing precision and diversity of today’s therapeutic landscape. Several approvals also opened doors in historically underserved areas, including non-opioid pain management, RSV prevention in infants, and dry eye disease. Together, these developments signal not just short-term progress, but a promising trajectory for the rest of 2025—and for the future of patient care across specialties.

To celebrate the first half of 2025, we have compiled a recap for each of the 16 novel drug approvals from January through June, including links to related coverage from HCPLive and other brands within the MJH Life Sciences family of brands, including OncLive, Contemporary Pediatrics, and Contemporary OB/GYN.

Novel Drug Approvals Recap: First Half 2024

1. Datopotamab deruxtecan-dlnk (Datroway)

Date: January 17, 2025

Indication: Unresectable or metastatic, HR-positive, HER2-negative breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease

Background: Approval based on data from the phase 3 TROPION-Breast01 trial, which showed patients treated with Dato-DXd (n = 365) experienced a median progression-free survival of 6.9 months (95% CI, 5.7-7.4) versus 4.9 months (95% CI, 4.2-5.5) for those given chemotherapy (n = 367; hazard ratio [HR], 0.63; 95% CI, 0.52-0.76; 2-sided P < .0001)

2. Treosulfan (Grafapex)

Date: January 21, 2025

Indication: In combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia and myelodysplastic syndrome

Background: Approval based on data from the phase 3 MC-FludT.14/L trial, which compared treosulfan plus fludarabine (n = 280) versus busulfan plus fludarabine (n = 290) as a preparative regimen for alloHCT. Findings showed the experimental regimen led to a 33% reduction in the risk of death in the overall population compared with the control regimen (HR, 0.67; 95% CI, 0.51-0.90). Treosulfan plus fludarabine reduced the risk of death by 27% in patients with AML (HR, 0.73; 95% CI, 0.51-1.06) and 36% in patients with MDS (HR, 0.64; 95% CI, 0.40-1.02).

3. Suzetrigine (Journavx)

Date: January 30, 2025

Indication: Moderate to severe acute pain

Background: Approval based on data from 2 randomized, double-blind, placebo- and active-controlled trials of acute surgical pain, with one trial conducted among patients following abdominoplasty and the other following bunionectomy. In both trials, suzetrigine demonstrated a statistically significant superior reduction in pain compared to placebo.

4. Mirdametinib (Gomekli)

Date: February 11, 2025

Indication: Neurofibromatosis type 1 who have symptomatic plexiform neurofibromas not amenable to complete resection

Background: Approval based on data from the phase 2 ReNeu trial, which showed mirdametinib generated a confirmed overall response rate of 41% (95% CI, 29%-55%) in adult patients (n = 58) and 52% (95% CI, 38%-65%) in pediatric patients (n = 56).

5. Vimseltinib (Romvimza)

Date: February 14, 2025

Indication: Symptomatic tenosynovial giant cell tumor for which surgical resection will potentially cause worsening functional limitation or severe morbidity

Background: Approval based on findings from the phase 3 MOTION trial, in which vimseltinib elicited a 40% (95% CI, 29%-51%) overall response rate at week 25 versus 0% (95% CI, 0%-9%) with placebo in the intent-to-treat population (difference, 40%; 95% CI, 29%-51%; P < .0001). The median duration of response (DOR) was not reached with vimseltinib. At an additional 6 months of follow-up, 85% of responders had achieved a DOR of ≥ 6 months, and 58% of responders had a DOR of ≥ 9 months.

6. Gepotidacin (Blujepa)

Date: March 25, 2025

Indication: Uncomplicated urinary tract infections caused by Escherichia coli, Klebsiella pneumoniae, Citrobacter freundii complex, Staphylococcus saprophyticus and Enterococcus faecalis in female adults weighing ≥40 kg and adolescents ≥12 years of age weighing ≥40 kg

Background: Approval based on positive results from the phase 3 Efficacy of Antibacterial Gepotidacin Evaluated (EAGLE)-2 and EAGLE-3 trials, where gepotidacin demonstrated non-inferiority to nitrofurantoin, the current standard of care for uUTI, in female adults and adolescents with a confirmed uUTI and a uropathogen susceptible to nitrofurantoin.

7. Fitusiran (Qfitlia)

Date: March 28, 2025

Indication: Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients 12 years of age and older with hemophilia A or hemophilia B, with or without factor VIII or IX inhibitors

Background: Approval based on data from 2 multicenter, randomized clinical trials which enrolled a total of 177 adult and pediatric male patients with either hemophilia A or hemophilia B. In the participants with inhibitors who received the antithrombin-based dosing regimen of Qfitlia, there was a 73% reduction in estimated annualized bleeding rate compared to those who received on-demand treatment with bypassing agents. In participants without inhibitors who received the antithrombin-based dosing regimen of Qfitlia, there was a 71% reduction in estimated annualized bleeding rate compared to those who received on-demand treatment with clotting factor concentrates.

8. Atrasentan (Vanrafia)

Date: April 2, 2025

Indication: Proteinuria reduction in adults with primary immunoglobulin A nephropathy at risk of rapid disease progression

Background: Approval based on 36-week data from the ALIGN trial, which showed use of atrasentan was associated with a 36.1% (95% CI, -44.6 to -26.4; P < .0001) reduction in UPCR at week 36 relative to placebo therapy, with reductions as early as the first 6 weeks of treatment.

9. Penpulimab-kcqx

Date: April 23, 2025

Indication: In combination with either cisplatin or carboplatin and gemcitabine, to treat adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma, or as a single agent while on or after platinum-based chemotherapy and at least one other prior line of therapy

Background: Approval based on data from the phase 3 Study AK105-304 showing patients treated with penpulimab plus chemotherapy achieved a median progression-free survival of 9.6 months (95% CI, 7.1-12.5) versus 7.0 months (95% CI, 6.9-7.3) in those given placebo plus chemotherapy (HR, 0.45; 95% CI, 0.33-0.62; 2-sided P < .0001).

10. Nipocalimab-aahu (Imaavy)

Date: April 29, 2025

Indication: Generalized myasthenia gravis

Background: Approval based on data from the 24-week double-blind, placebo-controlled phase 3 VIVACITY-MG3 trial, which showed patients on nipocalimab had a 4.70-point improvement in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) score. This was significantly more than the 3.35-point improvement in the placebo arm over weeks 22, 23, and 24 (P = .002).

11. Avutometinib and defactinib (Avmapki Fakzynja Co-Pack)

Date: May 8, 2025

Indication: KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) after prior systemic therapy

Background: Approval based on data from RAMP-201, which showed the doublet elicited a confirmed overall response rate of 44% (95% CI, 31%-58%), which comprised a complete response rate of 3.5% and a partial response rate of 40%. The duration of response ranged from 3.3 months to 31.1 months.

12. Telisotuzumab vedotin-tllv (Emrelis)

Date: May 14, 2025

Indication: Locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) with high c-Met protein overexpression after prior systemic therapy

Background: Approval based on data from the phase 2 LUMINOSITY trial, which showed patients with high c-MET protein overexpression treated with telisotuzumab vedotin (n = 84) achieved an overall response rate of 35% (95% CI, 24%-46%) and a median duration of response of 7.2 months (95% CI, 4.2-12).

13. Acoltremon (Tryptyr)

Date: May 28, 2025

Indication: Signs and symptoms of dry eye disease

Background: Approval based on data from COMET-2 and COMET-3, which both met their primary endpoints for the proportion of individuals with a ≥10 mm increase in unanesthetized Schirmer’s score. This endpoint reached statistical significance at Day 14, with 42.6% versus 8.2% of patients in COMET-2 and 53.2% versus 14.4% in COMET-3 (both P <.0001). These results were consistent through Day 90.

14. Clesrovimab-cfor (Enflonsia)

Date: June 9, 2025

Indication: Prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in neonates and infants who are born during or entering their first RSV season

Background: Approval based on data from the phase 2b/3 CLEVER trial (MK-1654-004) evaluating a single dose of clesrovimab administered to preterm and full-term infants as well as the phase 3 SMART trial (MK-1654-007) evaluating the safety and efficacy of ENFLONSIA versus palivizumab in infants at increased risk for severe RSV disease.

15. Taletrectinib (Ibtrozi)

Date: June 11, 2025

Indication: Locally advanced or metastatic ROS1-positive non-small cell lung cancer

Background: Approval based on data from the phase 2 TRUST-1 and TRUST-II trials. Treatment-naive patients experienced an overall response rate (ORR) of 90% (95% CI, 83%-95%) in TRUST-I and 85% (95% CI, 73%-93%) in TRUST-II. In patients previously treated with a TKI, the ORR was 52% (95% CI, 39%-64%) in TRUST-I and 62% (95% CI, 46%-75%) in TRUST-II.

16. Garadacimab-gxii (Andembry)

Date: June 16, 2025

Indication: Hereditary angioedema

Background: Approval based on results from the placebo-controlled phase 3 VANGUARD trial showing 62% of patients treated with garadacimab-gxii were attack-free during the treatment period, and HAE attacks in the garadacimab-gxii arm were reduced by ≥ 99% compared with placebo.