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FDA Drug Approvals | 2024 | iPharmaCenter

Updated: Apr 20

Sl No







Stage III-IVA Cervical cancer








Lung cancer
















Pulmonary Arterial Hypertension








ALK-positive NSCLC

January 12, 2024

FDA Approved Keytruda for with FIGO 2014 Stage III-IVA cervical cancer

The approval from the U.S. Food and Drug Administration (FDA) for using Merck's Keytruda (pembrolizumab) alongside chemoradiotherapy in treating patients diagnosed with FIGO 2014 Stage III-IVA cervical cancer is a notable development. This regulatory milestone signifies Keytruda as the inaugural anti-PD-1 therapy sanctioned for this specific combination.

This FDA approval marks the third acknowledgment of Keytruda's efficacy in cervical cancer and is the 39th overall approval for Keytruda in the United States.

Merck announced the FDA's approval for Keytruda's use in combination with chemoradiotherapy (CRT) for patients diagnosed with FIGO 2014 Stage III-IVA cervical cancer. The approval is based on the data derived from the Phase 3 KEYNOTE-A18 trial, where the combination of Keytruda and CRT exhibited a 41% risk reduction in disease progression or mortality compared to the placebo plus CRT for patients with FIGO 2014 Stage III-IVA disease. Median PFS remained undetermined in both study groups.

The KEYNOTE-A18, also identified as ENGOT-cx11/GOG-3047, is a multicenter, randomized, double-blind, placebo-controlled Phase 3 trial. Carried out in collaboration with the European Network for Gynaecological Oncological Trial (ENGOT) groups and the GOG Foundation, the trial enrolled 1,060 cervical cancer patients without any prior definitive surgery, radiation, or systemic therapy. Among them were 596 patients diagnosed with FIGO 2014 Stage III-IVA cervical cancer and 462 with FIGO 2014 Stage IB2-IIB cervical cancer. Patients were randomly assigned (1:1) to receive either Keytruda combined with cisplatin and radiotherapy or placebo with the same treatment regimen.

The trial demonstrated a statistically significant improvement in progression-free survival (PFS) for the overall study population. In an exploratory subgroup analysis for the 462 patients with FIGO 2014 Stage IB2-IIB disease, the PFS HR estimate was 0.91. Overall survival data were not matured at the time of PFS analysis.

In the subgroup analysis of 596 patients with FIGO 2014 Stage III-IVA disease, 61 patients (21%) in the Keytruda plus CRT arm experienced a PFS event compared to 94 patients (31%) in the placebo plus CRT arm. Median PFS was not reached in either arm. The 12-month PFS rate was 81% for Keytruda plus CRT versus 70% for placebo plus CRT.

The median exposure duration to Keytruda was 12.1 months, with fatal adverse reactions observed in 1.4% of patients receiving Keytruda in combination with chemoradiotherapy. Serious adverse reactions occurred in 30% of patients. Keytruda discontinuation due to adverse reactions occurred in 7% of patients, with diarrhea being the most common cause. Adverse reactions leading to the interruption of KEYTRUDA occurred in 43% of patients, with anemia, COVID-19, and decreased neutrophil count being the most frequently cited reasons.


February 16, 2024

FDA Approves First Cellular Therapy for Treating Unresectable or Metastatic Melanoma - Amtagvi

The United States Food and Drug Administration (FDA) has granted approval to Amtagvi, marking a significant advancement as the inaugural cellular therapy authorized for managing adult patients with a specific form of skin cancer known as melanoma, which cannot be surgically removed (unresectable) or has spread to other parts of the body (metastatic) after prior treatment with other therapies (a PD-1 blocking antibody, and if positive for BRAF V600 mutation, a BRAF inhibitor with or without a MEK inhibitor).

Melanoma, a type of skin cancer commonly triggered by exposure to ultraviolet light from sources such as sunlight or indoor tanning, while representing only about 1% of all skin cancers, contributes significantly to cancer-related fatalities. Failure to detect and treat melanoma early can result in metastatic disease, with the cancer spreading to other areas of the body.

Amtagvi received approval through the Accelerated Approval pathway, a mechanism through which the FDA may authorize drugs for severe or life-threatening diseases or conditions lacking adequate treatment options, provided the drug demonstrates an effect on a surrogate endpoint reasonably predictive of clinical benefit to patients (improving patient well-being or functionality, or extending survival). This pathway typically grants patients earlier access to a promising therapy while the company conducts further studies to confirm the anticipated clinical benefits. Ongoing confirmation trials are currently underway to validate Amtagvi's clinical efficacy.

The safety and effectiveness of Amtagvi were assessed through a global, multicenter, multicohort clinical study involving adult patients with unresectable or metastatic melanoma who had previously received at least one systemic therapy, including a PD-1 blocking antibody, and if positive for BRAF V600 mutation, a BRAF inhibitor or BRAF inhibitor with a MEK inhibitor. The efficacy was determined based on the objective response rate to treatment and the duration of response. Among the 73 patients treated with Amtagvi at the recommended dosage, the objective response rate was 31.5%, with three patients (4.1%) achieving a complete response and 20 patients (27.4%) showing a partial response. Among responders, 56.5%, 47.8%, and 43.5% maintained responses without tumor progression or death at six, nine, and twelve months, respectively.

Patients receiving Amtagvi may experience prolonged severe low blood count, severe infection, cardiac disorders, or may encounter worsened respiratory or renal function, potentially leading to fatal treatment-related complications. The drug's label carries a Boxed Warning highlighting these risks. Close monitoring for signs and symptoms of adverse reactions is crucial before and after infusion, with treatment adjustment or discontinuation warranted upon symptom manifestation.

The most commonly reported adverse reactions associated with Amtagvi include chills, fever, fatigue, tachycardia (abnormally fast heart rate), diarrhea, febrile neutropenia.


February 16, 2024

Tagrisso combined with chemotherapy garners approval in the United States for individuals with EGFR-mutated advanced lung cancer

AstraZeneca's Tagrisso (osimertinib) in conjunction with chemotherapy has gained regulatory approval in the United States for managing adult patients afflicted with locally advanced or metastatic non-small cell lung cancer (NSCLC) harbouring epidermal growth factor receptor-mutated (EGFRm) genes.

The green light from the Food and Drug Administration (FDA), following a Priority Review, hinged on the outcomes of the Phase III FLAURA2 trial, which were published in The New England Journal of Medicine.

Tagrisso coupled with chemotherapy demonstrated a 38% reduction in the risk of disease advancement or mortality compared to Tagrisso monotherapy, the prevailing first-line global standard of care.

Median progression-free survival (PFS), according to investigator evaluation, reached 25.5 months for patients treated with Tagrisso alongside chemotherapy, marking an enhancement of 8.8 months compared to Tagrisso monotherapy (16.7 months).

PFS findings from blinded independent central review (BICR) corroborated those from the investigator's assessment, illustrating a median PFS of 29.4 months with Tagrisso plus chemotherapy, signifying a 9.5-month advancement over Tagrisso monotherapy (19.9 months).

In a prearranged exploratory analysis of patients in the FLAURA2 trial exhibiting brain metastases at baseline, Tagrisso in combination with chemotherapy led to a 42% decline in the risk of central nervous system (CNS) ailment progression or mortality compared to Tagrisso alone as evaluated by BICR. Over a two-year follow-up, 74% of patients receiving Tagrisso with chemotherapy remained free from CNS ailment progression or mortality, in contrast to 54% of patients treated solely with Tagrisso.

Although overall survival (OS) findings remained premature at the second interim analysis (41% maturity), no indication of detriment was observed. The trial continues to monitor OS as a significant secondary endpoint.


March 01, 2024

FDA approved Janssen's Rybrevant as first-line therapy for EGFR 20 positive NSCLC

Amivantamab, marketed as Rybrevant, has gained FDA approval for its use alongside chemotherapy as a primary treatment option for patients diagnosed with non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations. This approval stems from findings in the Phase 3 PAPILLON trial, where the combination of Rybrevant with chemotherapy demonstrated a significant 61 percent decrease in the risk of disease progression or mortality compared to chemotherapy alone among previously untreated NSCLC patients with EGFR exon 20 insertion mutations.

The National Comprehensive Cancer Network (NCCN) has accordingly updated its clinical guidelines, endorsing the combination of amivantamab (RYBREVANT) and chemotherapy as a preferred initial therapy for NSCLC patients carrying EGFR exon 20 insertion mutations.

Previously granted accelerated approval in May 2021, the FDA's recent decision to fully approve RYBREVANT in conjunction with chemotherapy, specifically carboplatin-pemetrexed, for first-line treatment of locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations is rooted in the confirmatory Phase 3 PAPILLON trial results.

The FDA's decision was bolstered by positive outcomes from the Phase 3 PAPILLON study, which not only showcased a 61 percent reduction in the risk of disease progression or mortality with the combination therapy but also demonstrated improvements in objective response rate (ORR) and progression-free survival (PFS) compared to chemotherapy alone.

Conducted as a randomized, open-label Phase 3 trial, PAPILLON (NCT04538664) assessed the efficacy and safety of RYBREVANT in tandem with chemotherapy versus chemotherapy alone in newly diagnosed NSCLC patients with advanced or metastatic disease characterized by EGFR exon 20 insertion mutations. The study's primary endpoint focused on progression-free survival (PFS) as evaluated by blinded independent central review (BICR) using RECIST v1.1 guidelines, with secondary endpoints including objective response rate (ORR), PFS following initial subsequent therapy, time to symptomatic progression, and overall survival (OS). Patients initially treated with chemotherapy alone were permitted to transition to RYBREVANT monotherapy in the second-line setting upon confirmation of disease progression.


March 14, 2024

FDA Approved BMS'  Breyanzi for relapsed or refractory CLL or SLL

The U.S. FDA has given the green light to Bristol Myers Squibb’s Breyanzi, making it the leading CAR T cell therapy for adults facing relapsed or refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL). With its personalized approach delivered via a single infusion, Breyanzi represents a crucial breakthrough for patients previously lacking viable treatment options.

In the landmark TRANSCEND CLL 004 trial, Breyanzi's effectiveness in treating relapsed or refractory CLL or SLL patients was demonstrated. Notably, 20% of those treated with Breyanzi achieved complete response (CR), with the duration of this response proving promising. The approval of Breyanzi has been extended to include the treatment of relapsed or refractory large B-cell lymphoma and CLL or SLL, widening its scope to cater to a broader patient demographic.

Breyanzi, a CD19-directed chimeric antigen receptor (CAR) T cell therapy, is specifically intended for adult patients who have undergone at least two prior lines of therapy, including specific inhibitors such as Bruton tyrosine kinase (BTK) and B-cell lymphoma 2 (BCL-2) inhibitors. This regulatory approval has been granted under an accelerated pathway, contingent upon further validation from ongoing clinical trials.

The TRANSCEND CLL 004 trial, a multicenter study, has played a critical role in evaluating Breyanzi's efficacy.

The results have shown a notable complete response rate of 20%, with promising durability of response.

Additionally, high rates of minimal residual disease (MRD) negativity have been observed, indicating the therapy's efficacy.

Safety data from the trial have revealed primarily low-grade occurrences of cytokine release syndrome (CRS) and neurologic events (NEs), with manageable adverse events reported. Notably, the Phase 2 portion of the study is currently ongoing, focusing on evaluating the recommended dose of Breyanzi and its effectiveness in achieving a complete response rate, guided by established clinical guidelines.

The FDA's approval of Breyanzi marks a significant stride forward in CLL and SLL treatment, offering renewed hope for patients who have exhausted standard treatment options. Ongoing research will continue to validate its clinical benefits and further refine its utilization in patient care.


FDA Greenlights Novel Therapy for Duchenne Muscular Dystrophy

The U.S. Food and Drug Administration has granted approval for Duvyzat (givinostat), an oral medication intended for the treatment of Duchenne Muscular Dystrophy (DMD) in patients aged six years and above. Duvyzat marks a breakthrough as the first nonsteroidal drug sanctioned to address patients with all genetic variants of DMD. Functioning as a histone deacetylase (HDAC) inhibitor, it operates by selectively targeting pathological mechanisms to mitigate inflammation and muscle loss.

The effectiveness of Duvyzat in managing DMD underwent scrutiny through a rigorous 18-month Phase 3 study, conducted in a randomized, double-blind, placebo-controlled manner. The study's primary objective revolved around assessing the alteration in muscle function using a four-step stair climb as a benchmark. Throughout the study duration, all participants adhered to a standard steroid regimen, and upon completion of the 18-month treatment course, patients treated with Duvyzat exhibited a statistically notable deceleration in the decline of their four-step stair climb timing compared to the placebo group. The mean time change in climbing four stairs from baseline to the 18th month stood at 1.25 seconds for Duvyzat-treated patients in contrast to 3.03 seconds for those receiving the placebo.

Furthermore, a secondary efficacy metric encompassed evaluating the change in physical function from baseline to the 18th month, gauged through the North Star Ambulatory Assessment (NSAA), a widely employed scale for assessing motor function in ambulant boys with DMD. Remarkably, patients treated with Duvyzat demonstrated comparatively lesser deterioration in their NSAA scores after the 18-month intervention period.

Common adverse effects associated with Duvyzat usage include diarrhea, abdominal discomfort, reduced platelet count leading to heightened bleeding risk, nausea/vomiting, elevated triglyceride levels, and fever.

Prescribing guidelines for Duvyzat underscore the necessity for healthcare practitioners to assess patients' platelet counts and triglyceride levels before initiating treatment. Individuals with platelet counts below 150 x 109/L are advised against Duvyzat intake. Regular monitoring of platelet counts and triglyceride levels is recommended during the treatment course to ascertain the need for dosage adjustments. Moreover, instances of moderate to severe diarrhea may necessitate dosage modifications. Notably, Duvyzat administration bears the potential risk of QTc prolongation, which could predispose patients to irregular heart rhythms. Patients concurrently receiving medications associated with QTc prolongation or possessing specific types of heart conditions are cautioned against Duvyzat usage.

The prescribed dosage of Duvyzat is contingent upon the individual's body weight and should be administered orally twice daily alongside meals.


FDA Approves ELAHERE for Specific Ovarian Cancer Patients

The recent approval of Elahere stems from the conclusive MIRASOL Phase 3 trial, endorsing it as a potential new standard treatment for folate receptor alpha (FRα)-positive, platinum-resistant ovarian cancer (PROC). Evidence indicates that Elahere treatment yielded a survival advantage and mitigated the risk of cancer advancement by 35%.

Elahere marks AbbVie's maiden solid tumor treatment sanctioned subsequent to its recent acquisition of ImmunoGen.

The U.S. Food and Drug Administration (FDA) has fully approved Elahere (mirvetuximab soravtansine-gynx) for addressing folate receptor alpha (FRα)-positive, platinum-resistant epithelial ovarian, fallopian tube, or primary peritoneal adult cancer patients who have undergone up to three prior therapies.

These patients commonly present with advanced-stage disease, undergo surgical intervention, and subsequently receive platinum-based chemotherapy. However, resistance to this treatment may necessitate alternative therapies such as Elahere.

Elahere initially received FDA accelerated approval in November 2022, and the transition to full approval is grounded on data derived from the confirmatory Phase 3 MIRASOL trial. This trial juxtaposed Elahere against investigator's choice (IC) chemotherapy in patients with platinum-resistant ovarian cancer (PROC) exhibiting high FRα levels and having undergone up to three prior therapies. The primary endpoint of MIRASOL was progression-free survival (PFS) assessed by investigators, with key secondary endpoints including objective response rate (ORR) and overall survival (OS).

MIRASOL constitutes a randomized Phase 3 trial evaluating Elahere versus investigator's choice (IC) of monotherapy chemotherapy (weekly paclitaxel, pegylated liposomal doxorubicin, or topotecan). Eligibility criteria encompass patients with PROC featuring elevated FRα expression, as determined by the Ventana FOLR1 Assay, who have received up to three prior treatment regimens.

The primary endpoint of the trial is progression-free survival (PFS) assessed by investigators. Key secondary endpoints include objective response rate (ORR) and overall survival (OS).

The trial enrolled 453 participants, stratified by the number of previous lines of therapy (14% with one prior line, 39% with two prior lines, and 47% with three prior lines) and by IC chemotherapy, predominantly utilizing paclitaxel (41%), followed by PLD (36%) and topotecan (23%). Additionally, 62% of patients had previously received bevacizumab, while 55% had received a PARP inhibitor.

Based on current findings:

  • The hazard ratio (HR) for OS was 0.67, indicating a 33% reduction in the risk of death in the ELAHERE arm versus the IC chemotherapy arm

  • The HR for PFS was 0.65, signifying a 35% reduction in the risk of tumor or cancer progression in the Elahere arm compared to IC chemotherapy

  • Elahere exhibited fewer Grade 3+ adverse events overall and a lower discontinuation rate due to adverse events compared to the IC chemotherapy control group

The most prevalent (≥20%) adverse reactions, including laboratory abnormalities, comprised increased aspartate aminotransferase, fatigue, increased alanine aminotransferase, blurred vision, nausea, increased alkaline phosphatase, diarrhea, abdominal pain, keratopathy, peripheral neuropathy, musculoskeletal pain, decreased lymphocytes, decreased platelets, decreased magnesium, decreased hemoglobin, dry eye, constipation, decreased leukocytes, vomiting, decreased albumin, decreased appetite, and decreased neutrophils.


March 22, 2024

FDA Greenlights Opsynvi as Sole Once-Daily Tablet Combo Therapy for Pulmonary Arterial Hypertension (PAH)

Opsynvi marks a breakthrough by amalgamating two established treatments with proven efficacy and safety profiles into a once-daily tablet, aligning with clinical guidelines advocating early adoption of combination therapy.

Johnson & Johnson's extensive PAH lineup now encompasses remedies targeting all three essential and guideline-endorsed pathways for this rare, progressive ailment. Regulatory approval stems from the Phase 3 A DUE study, demonstrating significant enhancement in pulmonary hemodynamics, meeting its co-primary endpoints.

The U.S. Food and Drug Administration (FDA) has greenlit Opsynvi – a single-tablet fusion comprising macitentan, an endothelin receptor antagonist (ERA), and tadalafil, a phosphodiesterase 5 (PDE5) inhibitor – for the chronic management of adults grappling with pulmonary arterial hypertension (PAH, World Health Organization [WHO] Group I) and WHO functional class (FC) II-III. Opsynvi is suitable for treatment-naïve patients with PAH or those already receiving an ERA, PDE5 inhibitor, or both. Additionally, Opsynvi can be administered to patients concurrently receiving stable doses of macitentan 10 mg and tadalafil 40 mg (20 mg x 2) as separate tablets.

The FDA's endorsement of Opsynvi draws from the pivotal Phase 3 A DUE study, where Opsynvi exhibited superior reduction in Pulmonary Vascular Resistance (PVR) after 16 weeks compared to tadalafil or macitentan monotherapy. Opsynvi bears a Boxed Warning owing to embryo-fetal toxicity risk, necessitating female patients' enrollment in the Macitentan-Containing Products Risk Evaluation and Mitigation Strategy (REMS) program.

With this approval, Johnson & Johnson bolsters its PAH arsenal, encompassing therapies targeting nitric oxide, endothelin, and prostacyclin pathways, as recommended by foundational guidelines.


March 25, 2024

FDA Gives Green Light to Ultomiris for Treating Neuromyelitis Optica Spectrum Disorder (NMOSD) in Adults

Ultomiris (ravulizumab) has secured approval in the United States (US) as the premier and solitary long-lasting C5 complement inhibitor designated for adult patients grappling with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder (NMOSD).

The US Food and Drug Administration's (FDA) approval was grounded in the favorable outcomes from the CHAMPION-NMOSD Phase III trial, disclosed in the Annals of Neurology. In this trial, Ultomiris was pitted against an external placebo arm derived from the pivotal Soliris PREVENT clinical trial.

Ultomiris achieved the primary goal of time to first on-trial relapse, validated by an independent adjudication committee. Notably, Ultomiris patients exhibited zero relapses over a median treatment span of 73 weeks (relapse risk reduction: 98.6%).

NMOSD stands as a rare and incapacitating autoimmune disorder affecting the central nervous system (CNS), encompassing the spinal cord and optic nerves. The majority of NMOSD patients confront unpredictable relapses, marked by the onset of new neurological symptoms or exacerbation of existing ones, often severe and recurrent, potentially leading to enduring disability.

On the whole, the safety and tolerability profile of Ultomiris in the CHAMPION-NMOSD trial remained consistent with prior clinical investigations and real-world usage, devoid of any emergent safety concerns. The prevailing adverse events (AEs) encompassed COVID-19, headache, back pain, arthralgia, and urinary tract infection.


AstraZeneca aims to continue Soliris legacy with the approval of Voydeya

Voydeya has been approved in the US as an additional treatment alongside ravulizumab or eculizumab for treating extravascular haemolysis in adults with the rare condition PNH. The approval was based on the successful outcomes from the pivotal ALPHA Phase III trial.

Voydeya, also known as danicopan, has gained approval in the U.S. as supplementary therapy to ravulizumab or eculizumab for handling extravascular haemolysis (EVH) in adults grappling with paroxysmal nocturnal haemoglobinuria (PNH). This pioneering oral Factor D inhibitor has been designed to complement the standard-of-care medications Ultomiris (ravulizumab) or Soliris (eculizumab), aiming to cater to the specific requirements of approximately 10-20% of PNH patients facing clinically significant EVH despite undergoing treatment with a C5 inhibitor.

The endorsement from the U.S. Food and Drug Administration (FDA) was hinged upon affirmative outcomes emerging from the pivotal ALPHA Phase III trial.

The ALPHA Phase III trial meticulously assessed the efficacy and safety profile of Voydeya as an adjunct to Ultomiris or Soliris in PNH patients grappling with clinically significant EVH. The findings revealed that Voydeya satisfactorily achieved the primary endpoint, exhibiting a change in haemoglobin levels from baseline to week 12, along with meeting all critical secondary endpoints, encompassing transfusion avoidance and alterations in the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score.

Voydeya has secured Breakthrough Therapy designation from the U.S. FDA and PRIority MEdicines (PRIME) status from the European Medicines Agency. Additionally, it has been granted Orphan Drug Designation in the U.S., European Union (EU), and Japan for addressing PNH.

Voydeya has already received approval in Japan and has garnered a recommendation for approval in the EU. Regulatory assessments are presently underway in various other nations.


Roche's Alecensa is approved for patients with early-stage ALK-positive NSCLC who have undergone tumour resection

Roche has announced that the U.S. Food and Drug Administration (FDA) approved Alecensa (alectinib) as an adjuvant treatment for patients with early-stage ALK-positive non-small cell lung cancer (NSCLC) who have undergone tumour resection. This approval stems from the Phase III ALINA study, which investigated Alecensa's efficacy in reducing disease recurrence or death among patients with completely resected IB to IIIA ALK-positive NSCLC.

In the ALINA study, Alecensa exhibited an unprecedented 76% reduction in the risk of disease recurrence or death compared to platinum-based chemotherapy. Furthermore, an exploratory analysis indicated a notable improvement in central nervous system disease-free survival. The safety profile of Alecensa in this trial remained consistent with previous studies in the metastatic setting, with no unexpected safety concerns arising.

The approval of Alecensa marks a significant advancement, particularly for the approximately 5% of NSCLC patients worldwide who are ALK-positive. Moreover, the FDA's Real-Time Oncology Review pilot program facilitated an efficient review process, ensuring timely access to this promising therapy. International collaboration under the FDA's Project Orbis initiative further expedites access to Alecensa for patients across borders. The Phase III ALINA study data will also support submissions to other regulatory agencies worldwide, extending the reach of this important treatment option for early-stage ALK-positive NSCLC patients.


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