As September begins, let’s take a look back at the regulatory highlights from last month and forecast what’s coming up.Adaptimmune Therapeutics plc (ADAP) saw its Tecelra gain accelerated FDA approval on August 1, 2024, for treating adults with unresectable or metastatic synovial sarcoma, a rare cancer. This marks the first new treatment for this disease in over a decade and the first gene therapy for metastatic synovial sarcoma.On August 7, the FDA approved Purdue Pharma’s Zurnai, making it the first nalmefene hydrochloride auto-injector available for emergency treatment of known or suspected opioid overdose in individuals aged 12 and older.Another milestone came on August 9 with the FDA’s approval of ARS Pharmaceuticals’ (SPRY) neffy, the first nasal spray for treating anaphylaxis.To combat the ongoing COVID-19 variants, the FDA approved updated mRNA vaccines from Moderna Inc. (MRNA) and Pfizer Inc. (PFE) on August 22.Globally, the World Health Organization declared the Mpox outbreak a public health emergency on August 14. Subsequently, Emergent BioSolutions Inc.’s (EBS) smallpox vaccine ACAM2000 received FDA approval for expanded use to prevent Mpox disease in high-risk individuals on August 30.Looking ahead, let’s explore some biotech stocks awaiting FDA decisions this September.### Travere Therapeutics Inc. (TVTX)The FDA will decide on converting the accelerated approval of Travere Therapeutics’ Filspari to traditional approval on September 5, 2024. Initially approved to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression in February 2023, Filspari generated $27.1 million in net sales in Q2 2024, up from $3.46 million the previous year.IgA nephropathy, or Berger’s disease, is a rare progressive kidney condition characterized by immunoglobulin A (IgA) buildup in the kidneys, disrupting normal filtering and leading to blood and protein in the urine, along with a gradual loss of kidney function. Travere has a license agreement with Ligand Pharmaceuticals (LGND) and must make milestone payments upon achieving certain regulatory and sales goals.TVTX closed Friday at $9.47, up 1.18%.### Avadel Pharmaceuticals plc (AVDL)Avadel Pharmaceuticals is seeking to expand the FDA-approved uses of Lumryz to include treating cataplexy or excessive daytime sleepiness (EDS) in pediatric narcolepsy patients. A decision is expected on September 7, 2024. Lumryz received tentative approval for treating adult narcolepsy symptoms in July 2022 and full approval in May 2023. It launched in the U.S. on June 5, 2023, and generated $45.1 million in revenue in Q2 2024, up from $1.5 million in the same quarter of 2023.AVDL closed Friday at $15.17, up 1.88%.### Iterum Therapeutics plc (ITRM)The FDA panel will review Iterum Therapeutics’ resubmitted New Drug Application for oral Sulopenem on September 9, 2024. The drug is intended for treating uncomplicated urinary tract infections (uUTI) in adult women. This is the company’s second attempt after the FDA initially declined approval in July 2021, citing the need for additional clinical trials. Iterum addressed the concerns and resubmitted the application in April 2024. A final decision is expected on October 25, 2024.ITRM closed Friday at $1.18, up 4.42%.### Roche Holding AG (RHHBY.OB)Roche Holding AG awaits the FDA’s decision on its Ocrevus SC for treating relapsing multiple sclerosis and primary progressive multiple sclerosis, expected on September 13, 2024.### Ocrevus SC Injection and Co-formulation with ENHANZEOcrevus SC, an injectable form of the multiple sclerosis treatment Ocrevus, utilizes ENHANZE, a proprietary recombinant human hyaluronidase enzyme developed by Halozyme Therapeutics Inc. (HALO). This new formulation offers a rapid 10-minute injection compared to the original intravenous (IV) version, which was approved in the U.S. in 2017 for both relapsing and primary progressive forms of multiple sclerosis. While maintaining the twice-yearly dosing schedule of the IV version, Ocrevus SC aims to broaden accessibility to treatment centers lacking IV infrastructure or facing IV capacity constraints. RHHBY.OB closed the previous trading session at $42.33, experiencing a minor decline of 0.05%.### Vanda Pharmaceuticals’ Tradipitant for GastroparesisVanda Pharmaceuticals Inc. (VNDA) is currently awaiting an FDA decision on its lead drug candidate, Tradipitant, intended to alleviate gastroparesis symptoms. This condition, characterized by delayed gastric emptying, affects an estimated 6 million Americans with symptoms such as nausea, vomiting, bloating, and abdominal pain. If approved by September 18, 2024, Tradipitant would be the first novel gastroparesis treatment approved by the FDA in over four decades. Despite the ongoing review, the FDA has issued a preliminary notice highlighting deficiencies that currently preclude labeling discussions. VNDA’s stock concluded Friday’s trading at $5.29, reflecting a 1.34% increase.### Zevra Therapeutics’ Arimoclomol for Niemann-Pick Disease Type CAn FDA decision regarding Zevra Therapeutics Inc.’s (ZVRA) drug Arimoclomol, proposed for Niemann-Pick disease type C (NPC), is expected by September 21, 2024. NPC is a rare, progressive neurodegenerative disorder arising from mutations in the NPC1 or NPC2 genes. Symptoms encompass severe neurological issues affecting speech, cognition, swallowing, and motor skills. This marks the company’s second attempt at approval; the first submission in June 2021 was declined due to a need for additional data. Zevra resubmitted the application in December 2023 after addressing prior concerns. An FDA panel recently voted 11-5 in favor of approval. If approved, Arimoclomol would be the first treatment specifically designed to slow NPC progression. The drug was originally developed by LadRx Corp. (LADX.OB), sold to Orphazyme A/S (now Zevra Therapeutics) in 2011, and subsequently acquired by XOMA Corp. (XOMA) in 2023. ZVRA’s shares closed at $7.65 on Friday, up by 3.52%.### Heron Therapeutics’ Zynrelef Vial Access NeedleHeron Therapeutics Inc. (HRTX) is awaiting an FDA decision on its Prior Approval Supplement application for the Zynrelef Vial Access Needle (VAN) by September 23, 2024. Zynrelef, an extended-release combination of Bupivacaine and NSAID Meloxicam, is used for postoperative pain management and currently administered via a needle-free application into the surgical site. Approval of the VAN could streamline the aseptic preparation of Zynrelef, reducing withdrawal time from three minutes to between 20 and 45 seconds, thereby improving safety and adoption rates. HRTX’s stock ended Friday’s trading at $1.93.### Merck’s Expansion for KeytrudaMerck & Co., Inc. (MRK) is seeking FDA approval to expand the use of its cancer drug Keytruda for first-line treatment of unresectable advanced or metastatic malignant pleural mesothelioma, in combination with chemotherapy. The FDA decision is expected by September 25, 2024. Malignant pleural mesothelioma, originating in the lung linings, constitutes about 75% of mesothelioma cases. It is highly aggressive, with a five-year survival rate of merely 12%.**Keytruda**Keytruda has received FDA approval in the U.S. for a multitude of indications, including melanoma, metastatic non-small cell lung cancer, Merkel cell carcinoma, renal cell carcinoma, esophageal carcinoma, head and neck cancer, Hodgkin’s lymphoma, urothelial carcinoma, gastric cancer, cervical cancer, hepatocellular carcinoma, endometrial cancer, squamous cell carcinoma, breast cancer, and biliary tract tumors.*MRK closed Friday’s trading at $118.45, up 0.84%.*—**Bristol-Myers Squibb Co. (BMY)**The FDA is expected to make a decision on KarXT, an antipsychotic intended for the treatment of schizophrenia in adults, by September 26, 2024.Bristol-Myers Squibb acquired KarXT through its $14 billion purchase of Karuna Therapeutics earlier this year. PureTech Health plc (PRTC) was instrumental in founding Karuna and co-inventing the KarXT program.KarXT functions as a dual M1/M4 muscarinic acetylcholine receptor agonist within the central nervous system, aimed at improving the positive, negative, and cognitive symptoms of schizophrenia. Unlike current treatments, KarXT does not block dopamine receptors directly, offering a potentially innovative approach to schizophrenia treatment.If approved, KarXT would represent the first new mechanism in over 50 years for treating patients with schizophrenia.*BMY closed Friday’s trading at $49.95, up 0.46%.*—**Regeneron Pharmaceuticals Inc. (REGN)**Regeneron and Sanofi (SNY) have submitted an application for the FDA approval of Dupixent as an add-on maintenance treatment for certain adult patients with uncontrolled chronic obstructive pulmonary disease (COPD). The decision is expected by September 27, 2024.Dupixent is being co-developed by Regeneron and Sanofi under a global collaboration agreement. The drug is already approved in the U.S. for the treatment of atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis, eosinophilic esophagitis, and prurigo nodularis.*REGN closed Friday’s trading at $1,184.69, up 0.49%.*—**Sanofi (SNY)**Sanofi’s Sarclisa, in combination with a VRd regimen (bortezomib, lenalidomide, and dexamethasone), is under priority review by the FDA for the treatment of transplant-ineligible patients newly diagnosed with multiple myeloma. The decision is expected by September 27, 2024.If approved, this indication would be the third for Sarclisa. The drug, combined with pomalidomide and dexamethasone, received FDA approval in March 2020 for adult patients with multiple myeloma who had undergone at least two prior therapies, including lenalidomide and a proteasome inhibitor. In March 2021, Sarclisa combined with carfilzomib and dexamethasone was approved for adult patients with relapsed or refractory multiple myeloma who had received one to three prior lines of therapy.*SNY closed Friday’s trading at $56.26, down 0.12%.*The material has been provided by InstaForex Company – www.instaforex.com
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