FDA Rejects Nine Investigational Drugs in Q1 2024: Key Setbacks for Regeneron, Viatris, Venatorx, and Others
Mapi Pharma
Drug Developer
Regeneron
Biopharmaceutical Manufacturer
Viatris
Generic Drug Manufacturer
VenatoRx Pharmaceuticals
Developer of Novel Anti-Infective Drugs
Melinta Therapeutics
Antibiotic R&D and Production Company
Vanda Pharmaceuticals
Biopharmaceutical Company
Minerva Neuroscience
Biopharmaceutical Manufacturer
As the world's most authoritative and rigorously reviewed food and drug administration agency, the FDA’s evaluation process is extremely complex and its standards are exceptionally strict. Gaining FDA approval for market entry undoubtedly represents a very high threshold. In the first quarter of 2024, numerous innovative drugs failed to pass the FDA’s stringent review. Below is a list of rejected drugs compiled for you.
On March 25, Regeneron announced that its CD3/CD20 bispecific antibody drug Odronextamab targets relapsed or metastatic follicular lymphoma.（FL）The marketing application for diffuse large B-cell lymphoma was rejected by the FDA. Regeneron stated that the sole reason for the rejection was issues with the enrollment status of the confirmatory clinical trial. Notably, the FDA mentioned in the complete response letter（CRL）No issues were found with the drug in terms of efficacy, safety, trial design, labeling, or manufacturing.
This CD3/CD20 bispecific antibody drug has successfully received approval for relapsed/refractory follicular lymphoma and relapsed/refractory diffuse large B-cell lymphoma.（DLBCL）The approval applies to patients who have received two or more lines of systemic therapy. At the ASH conference in December, Regeneron reported Phase 2 clinical data for DLBCL, showing a significant response rate of 52%. Upon successful market entry, Odronextamab is expected to become the third CD20xCD3 bispecific antibody on the market, following Genentech's Lunsumio and Genmab/AbbVie's Epkinly, offering lymphoma patients more treatment options.
March 11, Viatris（Viatris）Jointly announced with Mapi Pharma that they have received FDA feedback on GA Depot(Glatiramer Acetate, 40mg)New Drug Application for Relapsing Multiple Sclerosis（NDA）The Complete Response Letter（CRL）Regarding the specific details of the FDA's rejection, the two companies did not disclose much, only stating that they are carefully reviewing the response letter to determine the "appropriate follow-up actions" for GA Depot 40.
The core component of GA Depot 40—Glatiramer Acetate, as an immunomodulator, was approved in the United States for the treatment of multiple sclerosis in 1996.（MS）, which was marketed under the brand name Copaxone. In June 2023, glatiramer acetate also successfully launched in China. The NDA for GA Depot is primarily based on positive results from a Phase III study, which aimed to evaluate GA Depot versus placebo in the treatment of relapsing multiple sclerosis.（RMS）Efficacy, Safety, and Tolerability from the Patient Perspective. Study results showed that, compared to the placebo group, the annual relapse rate in the GA Depot group was significantly reduced by 30.1%.（P=0.0066）`, this achievement undoubtedly provides strong support for the further development and application of GA Depot.`
On February 23, Venatorx Pharmaceuticals and Melinta Therapeutics jointly announced that the FDA had taken action on cefepime-taniborbactam.（cefepime-taniborbactam）This new drug application（NDA）Released the full response letter（CRL）Cefepime-taniborbactam as a β-lactam/β-lactamase inhibitor（BL/BLI）Combination antibiotics, currently under review, aim to treat complicated urinary tract infections in adults.（cUTI）Including potential treatments for acute pyelonephritis caused by susceptible Gram-negative microorganisms.
Cefepime-taniborbactam is an experimental intravenous injection（IV）β-Lactam/β-Lactamase Inhibitor（BL/BLI）Antibiotic combination aimed at treating complicated urinary tract infections in adults（cUTIs）, including pyelonephritis, as well as hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia（HABP/VABP）。
Complete Response Letter Issued by the FDA（CRL）Pointed out, new drug application（NDA）No issues were found in the clinical safety and efficacy data. Meanwhile, the FDA did not require further clinical trials to support the approval of cefepime-taniborbactam. However, the FDA did request additional information on chemistry, manufacturing, and controls.（CMC）Data in this area, as well as detailed information on drugs, testing methods, and production processes.
On March 6, Vanda Pharmaceuticals announced that it had received the FDA's response regarding Hetlioz.(Tasimelteon)New Drug Application for the Treatment of Insomnia（sNDA）The complete response letter. The FDA stated in the letter that apart from labeling and post-marketing requirements/commitments, other deficiencies were identified, thus the current sNDA cannot be approved. Vanda Pharmaceuticals is carefully reviewing the feedback from the FDA and evaluating the next steps for a response strategy.
Hetlioz, as a melatonin receptor agonist, was successfully approved in the United States in January 2014 for the treatment of Non-24-Hour Sleep-Wake Disorder. In December 2020, the drug achieved another breakthrough with its new indication – Smith-Magenis Syndrome.(SMS)Sleep Disorder at Night Also Receives FDA Approval.
On February 27, Minerva Neurosciences, Inc. announced that it had received feedback from the FDA regarding its New Drug Application.（NDA）——Complete Response Letter for Roluperidone in the Treatment of Negative Symptoms of Schizophrenia（CRL）, in which the FDA explicitly rejected the application for this new drug. Upon the release of this news, Minerva's stock price plummeted, with a staggering drop of 59%.
The FDA detailed the following four reasons for rejection in the CRL: First, although the MIN-101C03 study demonstrated statistical significance on the primary efficacy endpoint, the data is not yet sufficient to constitute conclusive evidence of Roluperidone's substantial effectiveness; second, the submitted NDA lacks relevant data regarding the concomitant use of Roluperidone with antipsychotic drugs; furthermore, the existing NDA materials fail to provide adequate evidence to demonstrate that Roluperidone has significant clinical benefits in improving negative symptoms of schizophrenia; finally, within the submitted safety data, patients receiving the proposed dose（64mg）The number of patients treated with Roluperidone for at least 12 months is insufficient, which has impacted the assessment of its long-term safety.
To address these deficiencies, the FDA has required Minerva to submit at least one additional positive, adequate, and well-controlled study to support the safety and efficacy of Roluperidone in treating negative symptoms. Minerva must also provide additional data to demonstrate the safety and efficacy of Roluperidone when used in combination with other antipsychotic medications, further supporting Roluperidone’s efficacy and establishing the long-term safety of the proposed dose.
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