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The FDA cleared eight novel drugs in May across oncology, cardiovascular medicine, hepatology, infectious disease, and anesthesia. Two were accelerated approvals resting on surrogate measures, and the anti-infective entries came with tightly drawn labels.
Veppanu: Arvinas and Pfizer bring a PROTAC degrader to ESR1-mutated breast cancer
On May 1, the FDA approved Veppanu (vepdegestrant) from Arvinas and Pfizer for adults with ER-positive, HER2-negative, ESR1-mutated advanced or metastatic breast cancer that has progressed after at least one line of endocrine therapy. Rigel Pharmaceuticals announced a deal on May 12 to license global rights for $70 million upfront and describes it as the first and only FDA-approved PROTAC.
Vepdegestrant binds the estrogen receptor at one end and an E3 ligase at the other, tagging the receptor for destruction. In VERITAC-2, median progression-free survival in the ESR1-mutated group was 5.0 months against 2.1 months on fulvestrant, with responses in 19 percent versus 4 percent. QTc prolongation carries a warning.
Beqalzi: BeOne secures an accelerated approval in mantle cell lymphoma
On May 13, BeOne Medicines secured accelerated approval for Beqalzi (sonrotoclax), a once-daily tablet for adults with relapsed or refractory mantle cell lymphoma after at least two lines of systemic therapy, including a BTK inhibitor. BeOne describes it as the first and only BCL-2 inhibitor approved in the U.S. for the disease.
Sonrotoclax binds BCL-2 and displaces the pro-apoptotic proteins it holds. In a single-arm trial of 103 adults, the overall response rate was 52 percent, with complete responses in 16 percent and a median duration of response of 15.8 months. Tumor lysis syndrome occurred in 7 percent of patients who followed the dose ramp-up, and continued approval may depend on a confirmatory trial.
Baxfendy: AstraZeneca adds aldosterone synthase inhibition to hypertension care
On May 15, AstraZeneca gained approval for Baxfendy (baxdrostat), a once-daily tablet used with other antihypertensive drugs in adults whose hypertension is not adequately controlled. It stops aldosterone being made, where mineralocorticoid receptor antagonists such as spironolactone act downstream at the receptor.
BaxHTN randomized 794 patients, every one of them on a diuretic, to baxdrostat 1 mg, 2 mg, or placebo. At Week 12, systolic pressure fell 15.7 mmHg on 2 mg and 14.5 mmHg on 1 mg against 5.8 mmHg on placebo, placebo-adjusted reductions of 9.8 and 8.7 mmHg. Potassium monitoring follows from suppressing aldosterone.
Hepcludex: Gilead brings a hepatitis delta treatment to the U.S.
On May 22, Gilead Sciences received accelerated approval for Hepcludex (bulevirtide-gmod), a once-daily 8.5 mg subcutaneous injection for adults with chronic hepatitis delta virus infection without cirrhosis or with compensated cirrhosis. The FDA's announcement called it the first FDA-approved treatment for the infection.
Bulevirtide blocks the bile-acid transporter that the hepatitis D and B viruses use to enter liver cells. In the MYR301 study, 48 percent of treated patients met a combined virologic and biochemical endpoint at Week 48, against 2 percent of a delayed-treatment control group. A boxed warning says stopping treatment can trigger severe flare-ups, and Gilead must verify clinical benefit in a post-marketing study.
Decnupaz: AbbVie adds a CD123 conjugate in a rare blood cancer
On May 27, the FDA approved Decnupaz (pivekimab sunirine-pvzy) from AbbVie for adults with blastic plasmacytoid dendritic cell neoplasm. The antibody-drug conjugate came from ImmunoGen, which AbbVie bought for $10.1 billion in a deal that closed in February 2024.
The antibody binds CD123 and releases a payload that alkylates DNA. In the single-arm CADENZA trial, 69.7 percent of 33 untreated patients reached complete remission or complete remission with residual skin abnormality, as did 15.7 percent of 51 patients with relapsed or refractory disease. A boxed warning covers hepatotoxicity, including fatal veno-occlusive disease.
Xocova: Shionogi gains a prevention-only label for its COVID-19 antiviral
On May 29, Shionogi won approval for Xocova (ensitrelvir), a five-day oral antiviral for post-exposure prophylaxis of COVID-19 in adults and adolescents 12 and older. The drug has been approved in Japan as a COVID-19 treatment since 2022, and the U.S. label covers prevention only.
Ensitrelvir inhibits the SARS-CoV-2 main protease. In SCORPIO-PEP, symptomatic infection occurred in 2.9 percent of household contacts on Xocova against 9.0 percent on placebo through day 10, a 67 percent reduction. The label carries an embryofetal toxicity warning and notes that the drug is a strong CYP3A inhibitor.
Cypsedo: Haisco brings a China-developed anesthetic to the U.S.
Also on May 29, the FDA approved Cypsedo (cipepofol) from Haisco Pharmaceutical Group, an intravenous anesthetic for the induction of general anesthesia in adults undergoing surgery. Haisco says it developed the drug in China, where it was already on the market.
Cipepofol is a general anesthetic that the label says is thought to act through the GABA-A receptor, with a mechanism that is otherwise poorly understood. It is given as an induction dose by a provider trained in inducing general anesthesia, immediately before surgery.
Zaynich: Wockhardt brings an in-house antibiotic combination to complicated urinary infections
Closing the month on May 29, Wockhardt gained approval for Zaynich (cefepime and zidebactam), an intravenous antibiotic for adults with complicated urinary tract infections, including pyelonephritis, caused by designated susceptible organisms. Wockhardt describes it as the first new chemical entity fully developed and commercialized by an Indian company to receive FDA approval.
The combination pairs a cephalosporin with zidebactam, which the label classes as both a beta-lactamase inhibitor and an antibacterial in its own right. In a non-inferiority trial that randomized 530 adults, combined clinical cure and microbiological response in the primary analysis population reached 89.0 percent on Zaynich against 68.4 percent on meropenem, a difference of 20.6 points. Warnings cover hypersensitivity, neurotoxicity, and Clostridioides difficile infection.
The Strategic Brief
Veppanu and Baxfendy are the market-shaping approvals. One brings targeted protein degradation into approved oncology practice, and the other moves the attack on aldosterone upstream of the receptor for patients already on other antihypertensives.
Beqalzi, Hepcludex, and Decnupaz are the narrow specialist launches. All three rest on response rates or surrogate markers, not survival outcomes, and two carry confirmatory obligations.
Xocova, Zaynich, and Cypsedo complete the month. The first two are anti-infectives with tightly drawn labels, and the third adds an induction agent in anesthesia.
Sources
Veppanu: FDA prescribing information; Drugs@FDA, NDA 219835; Onyx coverage.
Beqalzi: FDA prescribing information; Drugs@FDA, NDA 220711; Onyx coverage.
Baxfendy: FDA prescribing information; Drugs@FDA, NDA 219878; Onyx coverage.
Hepcludex: FDA prescribing information; Drugs@FDA, BLA 761468; Onyx coverage.
Decnupaz: FDA prescribing information; Drugs@FDA, BLA 761460; Onyx coverage.
Xocova: FDA prescribing information; Drugs@FDA, NDA 220442; Onyx coverage.
Cypsedo: Drugs@FDA, NDA 220482; FDA prescribing information; BioPharma APAC.
Zaynich: FDA prescribing information; Drugs@FDA, NDA 220787; Onyx coverage.
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