As we edge past the midpoint of 2025, the U.S. the oral solid dose sector has already put up some regulatory wins.
Last year, the Center for Drug Evaluation and Research (CDER) approved a total of 50 new drugs, 40% of which were OSD formulations — 14 tablets and six capsules.
How is 2025 shaping up? FDA shakeups withstanding, CDER has approved 23 novel drugs so far in 2025, 11 of which are oral solid dose formulations. Of the new OSD approvals, six are tablets, two are capsules, one is a powder, one is a tablet/capsule co-pack and one is approved in both capsule and tablet form.
Here’s a closer look at the 2025 OSD approvals.
Boehringer Ingelheim’s Hernexeos
Approval Date: August 8, 2025
The FDA granted accelerated approval to Boehringer Ingelheim’s oral kinase inhibitor, branded Hernexeos, to treat certain non-small cell lung cancer (NSCLC) patients. Hernexeos was approved to treat adults with unresectable or metastatic NSCLC whose tumors have HER2 (ERBB2) tyrosine kinase domain (TKD) activating mutations, as detected by an FDA-approved test, and who have received prior systemic therapy.
Approximately 2% to 4% of NSCLC cases have HER2 mutations. These cases often have a poor prognosis and a higher incidence brain metastases. The accelerated approval is based on data from the phase 1b Beamion-LUNG 1 trial, demonstrating an objective response rate of 75% (N=71).
PTC Therapeutics’ Sephience
Approval Date: July 28, 2025
PTC Therapeutics’ oral powder, branded Sephience, was approved for the treatment of children and adults living with phenylketonuria (PKU), a rare metabolic disease that affects the brain.
The approval includes labeling for the treatment of the broader condition, hyperphenylalaninemia — where there are elevated levels of the amino acid phenylalanine in the blood — in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU.
Sephience — an oral powder mixed with water or apple juice and administered once daily — is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase. The drug won marketing authorization from the European Commission in June 2025, greenlighting it in all 27 EU member states as well as Iceland, Norway and Liechtenstein.
KalVista Pharmaceuticals’ Ekterly
Approval Date: July 3, 2025
KalVista Pharmaceuticals’ novel plasma kallikrein inhibitor, branded Ekterly, was approved for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older.
Ekterly is the first and only oral on-demand treatment for HAE attacks — painful and debilitating tissue swelling in various locations of the body.
The approval comes after some FDA controversy. Back in mid-June, KalVista revealed that the agency notified the company on June 13 that the previously disclosed June 17 PDUFA goal date will not be met due to “heavy workload and limited resources.” Days after that news was shared, rumors began to swirl that FDA staff members involved with KalVista’s application had received directives from agency head Marty Makary to issue a complete response letter. Sources noted that the request was met with numerous objections and ultimately was withdrawn by Makary.
Dizal Pharmaceutical’s Zegfrovy
Approval Date: July 2, 2025
China-based Dizal Pharmaceutical was granted accelerated approval for its oral EGFR inhibitor, Zegfrovy, for locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon20ins.
Zegfrovy is an irreversible EGFR inhibitor with uniquely designed molecular structure targeting a wide spectrum of EGFR mutations with wild-type EGFR selectivity. It is the only approved targeted oral treatment for NSCLC with EGFR exon20ins — the third most common type of EGFR mutation in NSCLC. The once-daily tablets were granted accelerated approval in China in August 2023.
The FDA simultaneously approved Thermo Fisher Scientific's Oncomine Dx Express Test as a next-generation sequencing companion diagnostic for Zegfrovy to identify NSCLC patients with EGFR Exon20 insertions.
Nuvation Bio’s Ibtrozi
Approval Date: June 11, 2025
The FDA approved Nuvation Bio’s oral tyrosine kinase inhibitor, Ibtrozi, for the treatment of adult patients with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).
Ibtrozi capsules are designed to address some of the outstanding challenges of treating ROS1+ NSCLC, a rare and aggressive form of lung cancer. The drug has demonstrated high response rates with durable benefit and intracranial activity and is generally well tolerated, providing a new treatment option for patients with advanced ROS1+ NSCLC.
In addition to the U.S., Ibtrozi is approved in China and additional global filings are underway.
Verastem Oncology’s Avmapki Fakzynja Co-Pack
Approval Date: May 8, 2025
The FDA granted accelerated approval to Verastem Oncology’s Avmapki Fakzynja Co-Pack (avutometinib capsules; defactinib tablets) for the treatment of adult patients with a rare form of ovarian cancer, KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC).
The co-pack is the first and only FDA-approved medicine for this disease. The indication is approved under accelerated approval — ahead of the June 30 PDUFA date — based on the 44% overall response rate and duration of response in the phase 2 RAMP 201 clinical trial. Continued approval is contingent upon verification and description of clinical benefit in a confirmatory trial.
Novertis’ Vanrafia
Approval Date: April 2, 2025
The FDA granted accelerated approval for Novartis’ Vanrafia, a potent and selective endothelin A (ETA) receptor antagonist, for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression. IgAN is a progressive, rare kidney disease in which the immune system attacks the kidneys. Up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis, often requiring maintenance dialysis and/or kidney transplantation.
Vanrafia, administered as an oral tablet, was granted accelerated approval based on a prespecified interim analysis of the phase 3 ALIGN study measuring the reduction of proteinuria at 36 weeks compared to placebo. The drug’s continued approval is contingent upon the verification of clinical benefit from the ongoing phase 3 study evaluating whether Vanrafia slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at week 1361.
The eGFR data are expected in 2026 and intended to support traditional FDA approval.
GSK’s Blujepa
Approval Date: March 26, 2025
The FDA approved GSK’s gepotidacin, branded Blujepa, for the treatment of uncomplicated urinary tract infections (uUTIs), marking the first in a new class of oral antibiotics for uUTIs in nearly 30 years.
Blujepa, an oral triazaacenaphthylene antibiotic that inhibits bacterial DNA replication, is approved for the treatment of female adults and pediatric patients (≥12 years) with uUTIs caused by the following susceptible microorganisms: Escherichia coli, Klebsiella pneumoniae, Citrobacter freundii complex, Staphylococcus saprophyticus and Enterococcus faecalis. The drug is the result of a 2013 public-private partnership between GSK and the Biomedical Advanced Research and Development Authority (BARDA).
The approval is based on positive results from the pivotal phase 3 EAGLE-2 and EAGLE-3 trials which demonstrated non-inferiority to nitrofurantoin, one of the leading current standard of care options for uUTIs. New treatments are needed as the number of infections caused by drug-resistant bacteria are increasing, driving up treatment failure rates.
According to GSK, the U.S. commercial launch for Blujepa in uUTIs is planned for the second half of 2025.
Mirum Pharmaceuticals’ Ctexli
Approval Date: February 24, 2025
Mirum’s Ctexli tablets are the first and only treatment approved for adults with cerebrotendinous xanthomatosis (CTX), a rare and debilitating disease resulting from a deficiency of a key enzyme in the bile acid synthesis pathway.
CTX is characterized by fatty yellow nodules located in the connective tissues within the brain. As the clinical course progresses, irreversible neurological deterioration leads to premature death. Mirum’s tablets are made of chenodiol, also known as chenodeoxycholic acid, a naturally occurring bile acid that was originally approved decades ago for the treatment of radiolucent stones in the gallbladder.
The approval came in advance of Ctexli’s Prescription Drug User Fee Act (PDUFA) date, which was March 28.
Ono Pharmaceutical’s Romvimza
Approval Date: February 14, 2025
Ono Pharmaceutical’s kinase inhibitor, Romvimza, was approved for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection could potentially cause worsening functional limitation or severe morbidity.
Surgery is the main treatment option for TGCT, a rare, non-malignant tumor that forms within or near joints. But for the subset of patients whose condition is made worse by surgical removal, systemic treatment options are limited. Romvimza oral capsules — developed by Ono’s subsidiary, Deciphera Pharmaceuticals — provide a new therapeutic option. The drugs will compete with Daiichi Sankyo’s TGCT capsules, branded Turalio, which won FDA approval in 2019.
SpringWorks Therapeutics’ Gomekli
Approval Date: February 11, 2025
SpringWorks’ MEK inhibitor, Gomekli, was approved for the treatment of both adults and children with neurofibromatosis type 1 (NF1) who have symptomatic plexiform neurofibromas — tumors that grow along the peripheral nerve sheath and can cause severe disfigurement, pain and functional impairment — that can’t be removed through surgery.
Gomekli, available in both capsule form and as a tablet for oral suspension, is the first and only medicine approved for both adults and children with NF1, a rare genetic disorder caused by a mutation in the NF1 gene. With the approval, SpringWorks was granted a rare pediatric disease priority review voucher by the FDA.
Vertex’s Journavx
Approval Date: January 30, 2025
The approval of Vertex’s Journavx, an oral, non-opioid pain signal inhibitor for the treatment of adults with moderate-to-severe acute pain, marks the first new class of pain medicine approved in the U.S. in more than 20 years.
The twice-daily oral tablets reduce pain by targeting NaV1.8, a pain-signaling pathway involving sodium channels in the peripheral nervous system, before pain signals reach the brain. Importantly, because Journavx blocks pain signals only found in the periphery, not in the brain, it provides effective relief of pain without the limitations of currently available therapies, including the addictive potential of opioids.
Approvals to Watch
With numerous PDUFA dates already on the books, the FDA is weighing decisions that could shape treatment options across multiple therapeutic areas, including a non-hormonal treatment option for menopause and the first approved treatment for a rare childhood brain tumor.
Bayer’s elinzanetant
PDUFA Date: Within 90 days of July 26, 2025
Bayer’s elinzanetant is an investigational once-daily, non-hormonal treatment for moderate to severe vasomotor symptoms (VMS) associated with menopause.
The drug is the first dual neurokinin-1 and 3 (NK-1 and 3) receptor antagonist in late-stage clinical development for the non-hormonal treatment of VMS, also known as hot flashes, administered orally. Elinzanetant is thought to address VMS by modulating a group of estrogen sensitive neurons, called KNDy neurons, in the hypothalamus region of the brain.
In late July, the FDA extended the original PDUFA date of July 26 for up to 90 days. According to Bayer, the agency did not raise any concern regarding the general approvability of elinzanetant in its correspondence, but told the company that additional time was needed for a full review of the NDA submission, including information provided to support the application.
Elinzanetant was approved earlier in July by the UK’s MHRA, under the brand name Lynkuet.
Chimerix’s dordaviprone
PDUFA Date: August 18, 2025
Chimerix’s lead clinical asset, dordaviprone, is a novel first-in-class treatment in development for H3 K27M-mutant diffuse glioma, a rare, high-grade brain tumor that most commonly affects children and young adults.
If approved, the oral small molecule would the first FDA-approved therapies specifically for the condition; current standard of care is radiation. The agency has granted dordaviprone priority review and it is eligible for a rare pediatric disease priority review voucher.
Jazz Pharmaceuticals recently announced that it will acquire Chimerix for $935 million, picking up the dordaviprone asset in the deal.