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FDA Decisions Expected: August 2025

Your monthly synopsis of new drugs expected to hit the market 

July 16, 2025

Drug pipeline for August 2025

At Prime Therapeutics (Prime), we have positioned ourselves to best prepare our clients to manage new drugs. Our clinical and trade relations teams keep a keen eye on drugs awaiting approval by the United States (U.S.) Food and Drug Administration (FDA).
August 2025: denosumab (HLX14)
Organon and Henlius are awaiting FDA approval of HLX14, a biosimilar candidate for the RANK ligand (RANKL) inhibitor denosumab. The denosumab reference products are Prolia and Xgeva, by Amgen. Prolia is approved to treat osteoporosis or increase bone mass in select men and women who are at high risk for bone fracture. Xgeva is indicated for use in certain patients with cancers affecting the bone. If approved, HLX-14 will be the fifth denosumab biosimilar to be approved in the U.S. 
August 2025: doxecitine/doxribtimine
Doxecitine/doxribtimine was granted a Priority Review as well as Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease (RPD) designations for the treatment of thymidine kinase 2 deficiency (TK2d), an ultra-rare, debilitating and often life-threatening genetic mitochondrial disease characterized by progressive and severe muscle weakness. Doxecitine/doxribtimine is a combination pyrimidine nucleoside/nucleotide by UCB designed to increase or stabilize mitochondrial function leading to improved skeletal muscle function in patients with TK2d. Data from patients treated with pyrimidine nucleoside and/or nucleotide therapy (n=82) were gathered from retrospective and prospective sources and an expanded access program (EAP) and data from untreated patients (n=93) were from literature reviews of case reports and a retrospective chart review study.  Treatment with nucleoside and/or nucleotide therapy reduced risk of death by 92%–94% (hazard ratio [HR]=0.06–0.08; p<0.0001) and 87%–95% (HR=0.05–0.13; p<0.0001) in the time from symptom onset and starting treatment, respectively. Following treatment with pyrimidine nucleoside and/or nucleotide therapy, 75% of patients regained at least one previously lost motor milestone and 22.5% of patients regained four or more motor milestones. If approved, doxectine/doxribtimine will be the first medication indicated for the treatment of patients with TK2d.
Aug-Oct 2025: denosumab (Bmab-1000)
Biocon is awaiting FDA approval of Bmab-1000, a biosimilar candidate for the RANKL inhibitor denosumab. The denosumab reference products are Prolia and Xgeva, by Amgen. Prolia is approved to treat osteoporosis or increase bone mass in select men and women who are at high risk for bone fracture. Xgeva is indicated for use in certain patients with cancers affecting the bone. If approved, Bmab-1000 will be the sixth denosumab biosimilar to be approved in the U.S, if HLX-14 is approved first. 
Aug-Dec 2025: etripamil (Cardamyst)
Milestone submitted etripamil, a fast-acting, non-dihydropyridine calcium channel blocker (CCB), to the FDA for the acute conversion of atrioventricular (AV)-nodal-dependent paroxysmal supraventricular tachycardia (PSVT). This is the second full review for etripamil after the FDA issued a complete response letter (CRL) in March 2025 requiring additional information on nitrosamine impurities and facility inspection. The review time is expected to be within two or six months from the resubmission, depending on the classification. In the double-blind, placebo-controlled, Phase 3 RAPID trial, participants were trained to recognize onset of PSVT symptoms, attach an electrocardiogram (ECG) monitor and conduct the vagal maneuver. Enrollees were randomized to etripamil or placebo self-administered as an intranasal spray.² The study reported significantly more patients in the etripamil group experienced conversion of a PSVT episode to sinus rhythm within 30 minutes after the first dose compared to patients in the placebo group (64% versus 31%, respectively; p<0.0001). The median time to conversion was 17.2 minutes with etripamil compared to 53.5 minutes with placebo. Current guidelines by the American College of Cardiology, American Heart Association and Heart Rhythm Society (2015) advise that self-administered (“pill-in-the-pocket”) off-label use of acute doses of oral beta-blockers or non-dihydropyridine CCBs may be reasonable to terminate acute episodes of PSVT caused by AV-nodal re-entrant tachycardia in certain patients. If approved, etripamil will be the first agent specifically indicated for acute conversion of PSVT that can be self-administered.

For more information, see the etripamil (Cardamyst) Deep Dive in the January 2025 edition of Prime’s Quarterly Drug Pipeline. 
Aug. 1, 2025: elinzanetant 
Elinzanetant is a dual neurokinin (NK)-1 and NK-3 receptor antagonist submitted by Bayer to the FDA for the non-hormonal treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause. In the Phase 3, double-blind, randomized, placebo-controlled OASIS 1 and OASIS 2 trials, elinzanetant led to a statistically significant decrease in the primary endpoint of daily VMS frequency and severity from baseline to weeks 4 and 12 compared to placebo (p<0.001 for all) in postmenopausal participants aged 40 to 65 years experiencing moderate to severe VMS.³ At week 4, 62.8% and 62.2% of patients in the elinzanetant groups achieved at least a 50% reduction in VMS frequency in OASIS 1 and 2, respectively, compared with 29.2% and 32.3%, respectively, in the placebo groups. At week 12, 71.4% and 74.7% of patients in the elinzanetant groups achieved at least a 50% reduction in VMS frequency the OASIS 1 and 2, respectively, compared with 42.0% and 48.3%, respectively, in the placebo groups. The Phase 3 double-blind, randomized, placebo-controlled OASIS 3 trial demonstrated reduced VMS at 12 weeks compared to placebo (primary endpoint) and the VMS reductions were maintained for up to 52 weeks.⁴ In clinical trials, elinzanetant was administered orally once daily. If approved, it will be the first dual NK-1/-3 receptor antagonist for VMS in the U.S.
Aug. 8, 2025: aceclidine (LNZ100)
Lenz Therapeutics submitted LNZ100 for the treatment of presbyopia. It is a preservative-free, single-use, once-daily eye drop containing the muscarinic receptor agonist aceclidine. The Phase 3, vehicle-controlled CLARITY-2 trial reported, on day 1 of treatment, 71%, 71% and 40% of participants who received LNZ100 achieved a ≥ 3-line improvement in best corrected distance visual acuity (BCDVA) and no loss of 5 letters or more at 4 meters (primary endpoint) at 0.5, 3 and 10 hours, respectively, post dose compared with 12%, 8% and 5% of patients who received vehicle.⁵ In the Phase 3 CLARITY-1 trial, significantly more patients who received LNZ100 achieved this primary endpoint measure at the same timepoints (72%, 64% and 27%, respectively) compared to patients who received brimonidine (14%, 12% and 6%, respectively). If approved, LNZ100 will compete with the pilocarpine ophthalmic products Vuity and Qlosi for the treatment of presbyopia. 
Aug. 12, 2025: brensocatib 
Brensocatib, by Insmed and AstraZeneca, is an oral, reversible dipeptidyl peptidase 1 (DPP-1) inhibitor designed to reduce damage to the lungs by inhibiting DPP-1 in patients with non-cystic fibrosis bronchiectasis (NCFB), a chronic inflammatory lung disease. Brensocatinib received Breakthrough Therapy and Orphan Drug designations as well as a Priority Review from the FDA. In the double-blind, placebo-controlled Phase 3 ASPEN trial, once-daily oral doses of brensocatinib significantly reduced the primary endpoint of annualized rate of pulmonary exacerbations (placebo adjusted reduction, 21.1% [p=0.0019] with 10 mg dose and 19.4% [p=0.0046] with 25 mg dose) among patients with NCFB.⁶ Brensocatib also led to improvement in lung function. The Institute for Clinical and Economic Review (ICER) announced that it will assess the comparative clinical effectiveness and value of brensocatib for the treatment of NCFB and will discuss findings at a public meeting in September 2025.

For more information, see the brensocatib Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline
Aug. 18, 2025: rebisufligene etisparvovec (UX111) 
UX111 is an in vivo gene therapy that uses a self-complementary AAV9 vector to deliver a functional copy of the N-sulfoglucosamine sulfohydrolase (SGSH) gene to cells via a one-time intravenous (IV) infusion. Ultragenyx is seeking Accelerated Approval for UX111 for the treatment of Sanfilippo A syndrome (Mucopolysaccharidosis III type A [MPS-IIIA]), a rare, lysosomal storage disease caused by a mutation in the SGSH gene. The mutation causes an accumulation of heparan sulfate in lysosomes and ultimately leads to intellectual and motor deterioration and behavioral disturbances. UX111 is being evaluated in the ongoing Phase 1/2/3 Transfer A trial. At a mean follow-up of 36 months, the study reported a 66% (p<0.0001) reduction in the primary endpoint of heparan sulfate exposure in the cerebrospinal fluid of patients who received UX111.⁷ Improvements in cognitive function were also observed. The FDA has granted UX111 Fast Track, Orphan Drug, Regenerative Medicine Advanced Therapy (RMAT) and RPD designations as well as a Priority Review. If approved, UX111 will be the first pharmacological treatment for Sanfilippo A syndrome.

For more information, see the rebisufligene etisparvovec Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline
Aug. 18, 2025: dordaviprone (ONC201) 
Chimerix is seeking Accelerated Approval for dordaviprone, a first-in-class oral, blood-brain barrier penetrating imipridone, for the treatment of patients with recurrent H3 K27M-mutant diffuse glioma. The FDA granted dordaviprone a Priority Review and Fast Track, Orphan Drug and RPD designations. Pooled data from Phase 1 and Phase 2 trials and a compassionate use program in a total of 50 patients at least 2 years of age with recurrent H3 K27M-mutant diffuse glioma revealed dordaviprone revealed an overall response rate (ORR) of 28%, with no complete responses.⁸ The median time to response was 4.6 months (range, 1.6–15.9), median duration of response (DOR) was 10.4 months (range, 7.4–15.4) and disease control rate (DCR) was 40%. If approved, dordaviprone will be the first systemic therapy that has shown benefit in treating recurrent H3 K27M-mutant diffuse glioma.

For more information, see the dordaviprone Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline
Aug. 18, 2025: semaglutide injectable (Wegovy) 
Novo Nordisk’s semaglutide (Wegovy) for subcutaneous (SC) administration is undergoing a Priority Review from the FDA for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH), which is a fatty liver condition associated with obesity and type 2 diabetes. If approved, Wegovy will be the first glucagon-like peptide-1 (GLP-1) receptor agonist to treat MASH. Topline results from the placebo-controlled, Phase 3 ESSENCE trial demonstrated that 62.9% of patients who received once weekly semaglutide 2.4 mg via SC injection had improved steatohepatitis and 36.8% had improved liver fibrosis compared to 34.1% and 22.4%, respectively, of patients who received placebo.⁹ Semaglutide was granted Breakthrough Therapy designation from the FDA for the treatment of MASH. 

For more information, see the semaglutide injectable Deep Dive in the upcoming July 2025 edition of Prime’s Quarterly Drug Pipeline  
Aug. 19, 2025: vatiquinone 
PTC Therapeutics is awaiting the FDA’s decision for vatiquinone for the treatment of Friedreich's ataxia (FA).  FA is a genetic, progressive, neurodegenerative movement disorder caused by mutations in the frataxin (FXN) gene. FA is characterized by unsteady gait and fine limb movement. Vatiquinone is a first-in-class selective 15-Lipoxygenase (15-LO) inhibitor designed to alleviate cellular inflammation, oxidative stress due to mitochondrial dysfunction that is associated with FA. Vatiquinone, administered orally three times a day, was evaluated in the Phase 2/3 MOVE-FA trial in patients 7 years and older with FA.¹⁰ While the trial did not demonstrate a statistically significant improvement in the primary endpoint measure of Friedreich Ataxia Rating Scale (mFARS) score (difference relative to placebo, -1.61 points; p=0.14), a statistically significant effect of vatiquinone was reported for the pre-specified endpoint of mFARS upright stability subscale (difference relative to placebo, -1.26 points; p=0.021). In addition, the 72-week open-label extension period reported a 3.7-point improvement in the mFARS score compared to a matched natural history cohort, which translated to a 50% slowing in disease progression over three years. Vatiquinone received Fast Track and Orphan Drug designations and a Priority Review from the FDA. If approved, it will be the first therapy for pediatric patients 7 years and older with FA and provide an alternative option to omaveloxolone (Skyclarys) for those 16 years of age and older.

For more information, see the vatiquinone Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline
Aug. 21, 2025: donidalorsen 
Donidalorsen, by Ionis, is an antisense oligonucleotide agent that was submitted to the FDA for prophylaxis of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years of age and older. Donidalorsen targets prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of HAE. The Phase 3, double-blind, placebo-controlled OASIS-HAE trial reported an 81% lower monthly HAE attack rate with donidalorsen administered SC every 4 weeks compared to placebo during weeks one to 25 (p<0.001) of treatnent, and a 55% reduction compared to placebo when donidalorsen was administered every 8 weeks (p=0.004).¹¹ In the open-label extension OASISplus study, HAE attack rates continued to improve over time, resulting in 93% and 92% improvement from baseline measured at the start of the OASIS-HAE study with every 4 and 8 week dosing, respectively.¹² If approved, donidalorsen will be the first prekallikrein antisense oligonucleotide that is administered SC once every 4 or 8 weeks for prophylaxis of HAE attacks. It will compete with C1 esterase inhibitors (Haegarda [SC twice weekly] and Cinryze [IV twice weekly]) and plasma kallikrein inhibitors (lanadelumab [Takhzyro; SC every 2 or 4 weeks] and berotralstat (Orladeyo; orally once daily). 
Aug. 27, 2025: bevacizumab-vikg 
Outlook resubmitted bevacizumab-vikg, an ophthalmic formulation of the vascular endothelial growth factor (VEGF) inhibitor bevacizumab (Avastin), for the treatment of wet age-related macular degeneration (wet AMD). Intravitreal administration of VEGF inhibitors is first-line treatment for wet AMD. FDA approved VEGF inhibitors include aflibercept (Eylea and biosimilars), brolucizumab-dbll (Beovu), faricimab-svoa (Vabysmo) and ranibizumab (Lucentis and biosimilars, Susvimo ocular implant). Based on favorable data, bevacizumab, repackaged (unbranded) for intravitreal use, has been used off-label for wet-AMD. This is the second review of bevacizumab-vikg by the FDA following a CRL issued in 2023. Data to satisfy the CRL from the NORSE EIGHT trial did not demonstrate noninferiority of bevacizumab-vikg compared to ranibizumab at week 8 based on improvement in BCVA from baseline.¹³ However, a complete analysis at week 12 reported that anatomical response (e.g., central retinal thickness) was similar between the two agents. If approved, Outlook’s bevacizumab-vikg will be the first FDA-approved bevacizumab ophthalmic formulation available in the U.S.
Aug. 27, 2025: sodium dichloroacetate (SL1009) 
Saol Therapeutics is awaiting an FDA decision for sodium dichloroacetate oral solution for the treatment of pyruvate dehydrogenase complex deficiency (PDCD) – in May 2025, the FDA extended their review by three months. PDCD is a rare mitochondrial disorder of carbohydrate oxidation caused by mutations in the genes that encode pyruvate dehydrogenase complex that plays a role in energy production for proper cell function. PDCD is typically associated with neurologic manifestations (e.g., abnormal brain imaging, hypotonia, seizures, ataxia) and metabolic abnormalities (increased plasma pyruvate, lactic acidemia, metabolic acidosis). The age of onset and severity of disease symptoms vary widely, ranging from onset in the prenatal period or in infancy leading to death in early childhood to later in childhood onset survival into adulthood. Carbohydrate restricted (ketogenic) diets and co-factor (e.g., thiamine) supplementation have been used to manage PDCD and may provide benefit. Sodium dichloroacetate was granted Fast Track, Orphan Drug and RPD designations and a Priority Review from the FDA. A placebo-controlled, cross-over Phase 3 trial (NCT02616484) did not demonstrate statistically significance change in motor domains as measured by Observer Reported Outcome (ObsRO) survey (primary efficacy endpoint) during the double-blind portion of the trial.¹⁴⁻¹⁵ However, statistically significant improvement in motor function in the intent-to-treat population (p=0.002) was demonstrated with longer duration of therapy, including the open label extension (OLE) period. Sodium dichloroacetate significantly (p=0.006) decreased plasma lactate concentrations, a key secondary endpoint. In addition, treatment with sodium dichloroacetate improved survival (p=0.027) compared to appropriately matched controls. If approved, sodium dichloroacetate will be the first medicine available to treat PCDC.
Aug. 27, 2025: zopapogene imadenovec (PRGN-2012) 
Precigen is seeking Accelerated Approval for PRGN-2012 for the treatment of recurrent respiratory papillomatosis (RRP), a rare condition characterized by the development of wart-like lesions (papillomas) in the respiratory tract. RRP is caused by the human papilloma virus (HPV), which is estimated to be present in 75%–80% of adults who have not received an HPV vaccine. There is currently no cure for RRP. However, the use the HPV vaccine (Gardasil-9) has markedly reduced the number of new RRP cases. Surgical removal of the papillomas is the standard of care for RRP and the frequency varies among individuals. Medicines such as antivirals (e.g., acyclovir, ribavirin, cidofovir) and interferon have been used off-label to delay papilloma recurrence. PRGN-2012 is an off-the-shelf AdenoVerse gene therapy designed to elicit immune responses directed against cells infected with HPV 6 or HPV 11. If approved, PRGN-2012 will be the first and only FDA-approved pharmacologic treatment that addresses the underlying cause of RRP. In a Phase 1/2 clinical trial (NCT04724980), PRGN-2012 was administered SC on day 1, followed by surgical debulking of respiratory papillomatosis lesions, with subsequent PRGN-2012 doses administered on days 15, 43 and 85.¹⁶ The study reported that 51% of adults who received PRGN-2012 required no surgical intervention to control RRP during the 12 months following treatment (complete response). The FDA granted PRGN-2012 Breakthrough Therapy and Orphan Drug designations as well as a Priority Review.

For more information, see the zopapogene imadenovec Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline
Aug. 29, 2025: rilzabrutinib
Rilzabrutinib is an oral, reversible, covalent Bruton’s tyrosine kinase (BTK) inhibitor by Sanofi under FDA review for the treatment of immune thrombocytopenic purpura (ITP). Primary ITP is an autoimmune disease characterized by thrombocytopenia due to impaired platelet production and increased platelet destruction in the body. Rilzabrutinib received Fast Track and Orphan Drug designations from the FDA for the treatment of ITP. Rilzabrutinib was evaluated in the Phase 3 LUNA 3 trial in patients ≥ 12 years of age with persistent or chronic primary ITP and either a nondurable or inadequate response with or contraindication or intolerance to standard of care for ITP.¹⁷ In the trial’s 24-week double-blind period, 23% of patients who received twice daily oral doses of rilzabrutinib and 0% of those who received placebo (p<0.0001) achieved the primary endpoint of durable platelet response, defined as the proportion of participants to achieve platelet counts ≥ 50,000/μL for at least eight out of the last 12 weeks of the 24-week blinded treatment period in the absence of rescue therapy. Based on pooled data from the double-blind and 28-week open-label periods, 29% of patients who received rilzabrutinib achieved a durable platelet response at data cutoff. If approved, rilzabrutinib will be the first BTK inhibitor indicated for the treatment of ITP and will provide another oral option for the condition.

For more information, see the rilzabrutinib Deep Dive in the April 2025 edition of Prime’s Quarterly Drug Pipeline
Aug. 31, 2025: lecanemab-irmb (Leqembi) SC 
Eisai and Biogen are awaiting FDA decision for a SC formulation of the amyloid beta-directed antibody lecanemab-irmb (Leqembi) for once weekly maintenance dosing for the treatment Alzheimer’s disease in patients with mild cognitive impairment (MCI) or mild dementia stage of disease. The open-label extension phase of the Clarity AD trial included 72 patients who received Leqembi for the first time as a weekly SC injection and 322 patients who received Leqembi via biweekly IV administration in the Clarity AD core study followed by SC administration in this substudy.¹⁸ The substudy reported that SC administration led to 14% greater amyloid plaque removal compared IV administration. In addition, pharmacokinetic data revealed drug exposure (area under the concentration curve [AUC]) for weekly SC administration is 11% higher than the biweekly IV formulation. If approved, Leqembi will be the only treatment for Alzheimer’s disease that can be administered at home via SC injection using an autoinjector that delivers the dose over approximately 15 minutes. 

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References  
  1. Press release: https://www.ucb.com/newsroom/press-releases/article/new-data-on-investigational-therapy-for-thymidine-kinase-2-deficiency-presented-at-muscular-dystrophy-association-mda-2025-conference 
  2. RAPID trial: https://www.thelancet.com/action/showPdf?pii=S0140-6736%2823%2900776-6 
  3. OASIS 1 and OASIS 2 trials: https://jamanetwork.com/journals/jama/fullarticle/2822766 
  4. Press release, OASIS 3: https://www.bayer.com/en/us/news-stories/phase-iii-study-oasis-3#xd_co_f=ZmYwMThlZmEtNGVhZi00MTY1LTg2YTEtZjE2ZmNmOTc1OTQw~ 
  5. CLARITY 1 and CLARITY 2 trials: https://d1io3yog0oux5.cloudfront.net/_1b75a0055e0f2e21ec4f05c07a9d694b/lenztx/db/2271/21447/pdf/Clarity+Topline+Presentation+Slides+04022024+final+locked.pdf 
  6. Press release: https://investor.insmed.com/2024-05-28-Insmed-Announces-Positive-Topline-Results-from-Landmark-ASPEN-Study-of-Brensocatib-in-Patients-with-Bronchiectasis 
  7. Press release: https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-new-data-demonstrating-treatment-ux111-aav 
  8. Press release: https://ir.chimerix.com/news-releases/news-release-details/chimerix-announces-updated-phase-2-response-assessment 
  9. ESSENCE trial: https://www.nejm.org/doi/10.1056/NEJMoa2413258?url_ver=Z39.88-2003 
  10. MOVE-FA trial: https://ir.ptcbio.com/static-files/13448bcb-111c-4c98-a716-d1ed989a7290 
  11. OASIS-HAE trial: https://www.nejm.org/doi/full/10.1056/NEJMoa2402478 
  12. Press release: https://www.prnewswire.com/news-releases/ionis-presents-positive-results-from-oasis-hae-and-oasisplus-studies-of-investigational-medicine-donidalorsen-in-patients-with-hereditary-angioedema-302160162.html 
  13. Press release: https://www.globenewswire.com/news-release/2025/02/28/3034713/0/en/Outlook-Therapeutics-Re-Submits-Biologics-License-Application-for-ONS-5010-as-a-Treatment-for-Wet-AMD-to-the-U-S-Food-and-Drug-Administration.html 
  14. Press release: https://www.prnewswire.com/news-releases/saol-therapeutics-announces-poster-presentation-at-the-umdf-mitochondrial-medicine-2025-conference-302486196.html   
  15. Press release: https://saolrx.com/saol-therapeutics-announces-submission-of-new-drug-application-nda-to-the-u-s-fda-for-sl1009/ 
  16. NCT04724980: https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(24)00368-0/abstract 
  17. Press release: https://www.globenewswire.com/news-release/2024/12/07/2993342/0/en/Press-Release-ASH-rilzabrutinib-demonstrated-significant-patient-benefit-in-the-first-positive-phase-3-study-of-a-BTK-inhibitor-in-ITP.html 
  18. Press release: https://www.eisai.com/news/2023/news202368.html#xd_co_f=ZmYwMThlZmEtNGVhZi00MTY1LTg2YTEtZjE2ZmNmOTc1OTQw~ 
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