LONDON (dpa-AFX) - July is observed as Sarcoma Awareness Month. Sarcoma is a rare type of cancer that affects muscles, fat, nerves, and other connective tissues. About 15,000 people in the U.S. are diagnosed with this type of cancer every year, according to reports. The theme for this year's Sarcoma Awareness Month is 'Know your normal. Act on your symptoms. Advocate for answers'.
There are several approved drugs in the U.S. for treating sarcomas. Adaptimmune Therapeutics' Tecelra, approved by the FDA in August 2024, for synovial sarcoma, a type of aggressive soft tissue sarcoma, is the latest sarcoma drug to reach the market.
As we get ready to welcome July, it's also time to track the important FDA decisions lined up for the month.
Dizal
The FDA decision on Sunvozertinib, an oral EGFR inhibitor developed by Dizal, a spinout of AstraZeneca plc (AZN), is due in July.
Sunvozertinib is proposed for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins), as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.
The drug was granted accelerated approval in China in 2023, making it the world's first and only oral treatment for NSCLC patients with EGFR exon 20ins.
Regeneron Pharmaceuticals Inc. (REGN)
Regeneron Pharma has two oncology therapeutics at the FDA altar, Linvoseltamab and Odronextamab, for which decisions are expected.
Linvoseltamab is a bispecific antibody designed to target BCMA (B-cell maturation antigen), found on the surface of myeloma cells, and CD3, a protein on T-cells that enables the immune system to recognize and attack cancer cells. This compound is proposed for the treatment of adult patients with relapsed or refractory multiple myeloma.
The initial Biologics License Application (BLA) for Linvoseltamab was turned down by the FDA in August 2024, due to issues at a third-party fill and finish manufacturing facility.
Following the resolution of these manufacturing concerns, Regeneron resubmitted the BLA earlier this year and now awaits the U.S. regulatory agency's decision, which is due on July 10, 2025.
Linvoseltamab received conditional marketing authorization in the European Union in April 2025 for the treatment of adults with relapsed and refractory (R/R) multiple myeloma.
The company's Odronextamab, proposed for the treatment of relapsed/refractory follicular lymphoma after two or more lines of systemic therapy, is also having a second go-around with the FDA.
The FDA denied approval for Odronextamab in March 2024, not due to clinical efficacy, safety, or manufacturing concerns, but rather issues related to the enrollment status of a confirmatory trial. Following achievement of the FDA-mandated enrollment target for the confirmatory trial, Regeneron resubmitted the BLA in February 2025, with a decision now expected by July 30, 2025.
REGN closed Friday's (June 27, 2025) trading at $521.
Otsuka Holdings Co. Ltd. (OTSKY.OB)
An FDA advisory committee is scheduled to review a supplemental New Drug Application for REXULTI, submitted by Otsuka Pharmaceutical on July 18, 2025. The application seeks approval for the use of REXULTI in combination with the antidepressant Sertraline for the treatment of adults with post-traumatic stress disorder (PTSD).
REXULTI, an atypical antipsychotic, is already approved in the U.S. for the treatment of major depressive disorder, schizophrenia, and agitation associated with dementia due to Alzheimer's disease.
Discovered by Otsuka, REXULTI is being co-developed by Otsuka and Lundbeck.
OTSKY.OB closed Friday's trading at $24.80, up 0.61%.
Roche (RHHBY.OB)
Roche, which is seeking to expand the U.S. label for its bispecific antibody therapy, Columvi, awaits the FDA decision on July 20, 2025.
The proposed label expansion would allow the use of Columvi in combination with gemcitabine and oxaliplatin (GemOx) for the treatment of relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) in patients who have received at least one prior line of therapy and are ineligible for autologous stem cell transplant (ASCT).
In a phase III trial, dubbed STARGLO, the Columvi combination showed a statistically significant and clinically meaningful 41% survival benefit in R/R DLBCL. The STARGLO study was a multiregional clinical trial (MRCT) that enrolled 274 patients globally across 62 sites in 13 countries, including the US, Australia, and multiple European countries, with the majority of patients (52%) enrolling outside of Asia.
Last month, an FDA panel, which reviewed the Columvi combination for people with relapsed or refractory diffuse large B-cell lymphoma, declined to recommend its approval, citing concerns about the applicability of the STARGLO data to the U.S. population. The panel emphasized the need for additional data, given that the majority of trial participants were not enrolled in the U.S.
Columvi monotherapy was previously granted accelerated approval in the U.S. in June 2023 for the treatment of adults with certain types of diffuse large B-cell lymphoma (DLBCL) or large B-cell lymphoma (LBCL) that has come back (relapsed) or that did not respond to previous treatment (refractory), and who have received 2 or more prior treatments for their cancer.
RHHBY.OB closed Friday's trading at $40.91, down 0.78%.
Replimune Group Inc. (REPL)
Replimune's Biologics License Application for RP1 in combination with Bristol-Myers Squibb Co.'s (BMY) blockbuster drug Opdivo (nivolumab), proposed for the treatment of patients with advanced melanoma, is under priority review by the FDA, with a decision expected on July 22, 2025.
RP1, also known as Vusolimogene Oderparepvec, is a modified form of the herpes simplex virus designed to selectively infect and kill cancer cells. In addition to direct tumor cell destruction, it stimulates the immune system by producing granulocyte-macrophage colony-stimulating factor (GM-CSF), a protein that enhances anti-tumor immune responses.
According to the company, RP1 is administered via intratumoral injection, delivering the treatment directly into the tumor, allowing for targeted delivery of the therapy to cancer cells.
REPL closed Friday's trading at $9.33, down 1.37%.
GSK plc (GSK)
GSK's Biologics License Application for Blenrep in combinations with bortezomib plus dexamethasone and pomalidomide plus dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior line of therapy is under FDA review, with a decision expected on July 23, 2025.
Blenrep as a monotherapy, was granted accelerated approval in the U.S. in August 2020 for the treatment of adult patients with relapsed or refractory multiple myeloma who had received at least four prior therapies. However, following the failure of a confirmatory trial to verify the clinical benefit demonstrated by interim data supporting Blenrep's tentative approval, the company withdrew the drug from the market in 2022.
Before the FDA makes a final decision on Blenrep on July 23, an FDA panel is scheduled to scrutinize the Blenrep combinations on July 17, 2025.
GSK closed Friday's trading at $38.07, down 1.48%.
Swedish Orphan Biovitrum AB (publ) (SWOBY.OB)
Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB, has sought FDA approval for the expanded use of DOPTELET for the treatment of thrombocytopenia in pediatric patients one year and older with persistent or chronic immune thrombocytopenia (ITP) who have had an insufficient response to a prior therapy.
The US regulatory agency's decision is expected on July 24, 2025.
DOPTELET, an orally administered thrombopoietin receptor agonist (TPO-RA), received its initial FDA approval in May 2018 for the treatment of thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo a procedure. In June 2019, its indication was expanded to include the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had an insufficient response to a previous treatment.
SWOBY.OB closed Friday's trading at $14.50.
Ascendis Pharma A/S (ASND)
The FDA decision on the expanded use of Ascendis Pharma's Skytrofa for the treatment of adults with growth hormone deficiency is expected on July 27, 2025.
Skytrofa, a human growth hormone, is already approved for the treatment of pediatric patients 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone. The drug, administered once weekly via a subcutaneous injection using the Skytrofa autoinjector, typically in the abdomen, thigh, or buttocks, received its initial FDA approval in August 2021.
The annual sales of Skytrofa were €202 million (excluding deductions) in 2024, compared to €178.6 million in 2023.
In 2023, Ascendis Pharma entered into a royalty funding agreement with Royalty Pharma plc (RPRX). As part of the deal, Ascendis received an upfront payment of $150 million in return for granting Royalty Pharma a 9.15% royalty on U.S. net sales of Skytrofa, starting from January 1, 2025.
ASND closed Friday's trading at $172.22, down 0.42%.
Apellis Pharmaceuticals Inc. (APLS)
Apellis Pharma's supplemental Biologics License Application seeking approval for a second indication of its drug EMPAVELI is under priority review by the FDA, with a decision expected on July 28, 2025.
The proposed new indication is for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), two severe and rare kidney diseases, which can lead to kidney failure.
EMPAVELI is administered twice weekly by subcutaneous infusion, either through a commercially available infusion pump or the proprietary Empaveli Injector. The drug was first approved by the FDA in May 2021 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare, chronic blood disorder.
The U.S. net product revenue of EMPAVELI was $98.1 million for the full year 2024, compared to $91 million in 2023.
APLS closed Friday's trading at $17.43, down 5.27%.
PTC Therapeutics Inc. (PTCT)
The FDA is scheduled to announce its decision on PTC Therapeutics' investigational drug, Sepiapterin, proposed for the treatment of pediatric and adult patients with phenylketonuria, on July 29, 2025.
Phenylketonuria (PKU) is a rare, inherited metabolic disease caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine, an essential amino acid found in all proteins and most foods. If left untreated or poorly managed, phenylalanine can build up to harmful levels in the body, leading to serious and irreversible complications, including permanent intellectual disability, seizures, developmental delays, memory impairment, and behavioral or emotional issues.
Sepiapterin has a dual mechanism of action to increase the activity of the phenylalanine hydroxylase (PAH) enzyme, which helps lower blood phenylalanine (Phe) levels.
BioMarin's oral drug Kuvan and injectable drug Palynziq are the two FDA-approved treatments for phenylketonuria.
PTCT closed Friday's trading at $49.84, up 0.59%.
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