Latest news with #ODAC
Medscape
6 days ago
- Business
- Medscape
DLBCL: FDA Rejects BLA for Second-Line Glofitamab Combo
The FDA rejected a supplemental Biologics License Application (sBLA) for glofitamab-gxbm (Columvi, Genentech) in combination with gemcitabine and oxaliplatin for the second-line treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL) in patients who are not candidates for autologous stem cell transplant. A complete response letter from the FDA stated that Genentech's phase 3 STARGLO trial did not provide sufficient evidence to support the proposed second-line DLBCL indication for the T cell engaging bispecific antibody in the US, according to a Genentech statement. However, a conditional FDA approval in the third-line or greater setting remains in place. FDA's decision regarding second-line glofitamab aligns with an advisory committee vote in May, which cited a lack of US patient representation in the trial. The Oncologic Drugs Advisory Committee (ODAC) voted 8-1 against recommending approval, as reported by Medscape Medical News . The FDA asked for ODAC review over concerns that nearly half of the STARGLO participants were from Korea, Taiwan, and China, with most of the remaining participants coming from Europe and Australia. Only 25 US patients were included. Outcomes among Asian vs non-Asian patients differed, with a strong trend toward worse overall survival (OS), rates in White patients and patients from Europe and the US, despite a strong overall 0.59 hazard ratio for OS. Similar trends were observed for progression-free survival and complete response rates. At the time, the FDA said the low US enrollment 'limits the agency's ability to assess the applicability of the study results to a US patient population,' and cited additional concerns regarding comparator drugs used in the trial, according to meeting documents. 'While we are disappointed with this outcome, we remain confident in the data supporting the value of Columvi for US patients who have relapsed following initial treatment, and its key role as monotherapy in the third-line setting,' Genentech's Chief Medical Officer and head of Global Product Developments, Levi Garraway, MD, PhD, said in the company statement regarding the sBLA rejection. 'We are committed to bringing Columvi to more people living with lymphoma and are actively exploring its potential in additional treatment settings, including as frontline therapy.' 'For patients with this aggressive form of lymphoma, effective treatment after relapse is paramount,' added Jeremy Abramson, MD, the principle STARGLO investigator and director of the Jon and Jo Ann Hagler Center for Lymphoma at the Massachusetts General Hospital Cancer Center, Boston. Abramson stressed that in the STARGLO trial, the glofitamab combination therapy improved OS, which 'could have a positive impact for patients earlier in their treatment journey.' The regimen is currently approved for this indication in more than 35 countries. It is approved in more than 60 countries for third-line or greater treatment of DLBCL, including in the US where the FDA granted accelerated approval in 2023. STARGLO was also intended as a post-marketing confirmatory study to support conversion of the accelerated approval to full approval, Genentech noted. Results were published in 2024 in The Lancet, and 2-year follow-up data showing sustained overall improvements in the primary and secondary endpoints were presented at 2025 American Society of Clinical Oncology Meeting. Genentech said the regimen is currently being investigated in combination with other agents as a first-line treatment for DLBCL in the phase 3 SKYGLO study.
Yahoo
18-07-2025
- Business
- Yahoo
GSK's Blenrep faces setback as FDA cancer committee votes against approval
The US Food and Drug Administration's (FDA's) Oncologic Drugs Advisory Committee (ODAC) has voted against the benefit/risk profile of GSK's Blenrep (belantamab mafodotin) just days before the drug's Prescription Drug User Fee Act (PDUFA) date. This marks a significant setback for the UK-based pharma company, which is aiming to regain approval of the therapy after it was pulled from the US market in 2022 due to concerns raised by late-stage trial data. Blenrep was approved under the FDA accelerated approval process in 2020 to treat multiple myeloma (MM). It is an antibody drug conjugate (ADC) that combines a humanised BCMA monoclonal antibody with the cytotoxic agent auristatin F, linked together by a non-cleavable linker. The ODAC committee had a 7–1 split against the combination of Blenrep plus pomalidomide and steroid dexamethasone and a 5-3 split against the combination of Blenrep with bortezomib and dexamethasone. GSK states it remains confident in the benefit/risk profile of Blenrep as a combination therapy and will continue to work closely with the FDA as they complete the review of the drug in patients with relapsed or refractory multiple myeloma (r/r MM). The FDA will consider the recommendation of the committee as it finalises its review on Blenrep in advance of the 23 July PDUFA date. The concern of ocular toxicity was first highlighted in a document published ahead of the ODAC meeting that took place on 17 July. The recommendation given by ODACs is usually followed by the FDA when deciding if a drug should be approved. Data supporting the drug's approval comes from the DREAMM-7 (NCT04246047) and DREAMM-8 (NCT04484623) studies. In the studies, many patients experienced keratopathy and Visual Acuity (KVA) events. All grade KVAs occurred in 92% and 93% of patients in DREAMM-7 and DREAMM-8, respectively, while more serious grade 3-4 events were found in 77% and 78% of patients in the same two studies. Blenrep combinations are approved in r/r MM in the UK and Japan, as well as other markets, including Switzerland, based on the results of DREAMM-8. Applications for approval in the European Union (EU) and China are ongoing, based on the results of the DREAMM-7 study. "GSK's Blenrep faces setback as FDA cancer committee votes against approval" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
17-07-2025
- Business
- Yahoo
GSK's Blenrep US comeback hindered by eye safety concerns
GSK's plan to bring blood cancer drug Blenrep (belantamab mafodotin) back to markets has hit a stumbling block, after the US Food and Drug Administration (FDA) identified eye safety concerns ahead of a meeting to discuss the therapy's benefit-risk ratio. FDA reviewers flagged high rates of ocular toxicity, including corneal disease, known as keratopathy, and visual acuity changes. Experts also identified poor tolerability of regimens and resulting uncertainty regarding proposed dose, as per a briefing document published on 15 July. Shares in London-listed GSK closed 1.19% down to a price of £1,402.5 following the document release compared to the market open on 15 July. Prices had not recovered by the market open on 16 July. The British drugmaker has a market cap of £57bn. The briefing document was published ahead of an Oncologic Drugs Advisory Committee (ODAC) on 17 July that will review data for Blenrep's safety and effectiveness. The recommendation given by ODACs is usually followed by the FDA when deciding if a drug should be approved. FDA reviewers wrote: 'While ocular adverse events have been seen with other antibody-drug conjugates used for the treatment of cancer, this toxicity is unique among therapies for the treatment of multiple myeloma.' The key trials supporting the drug's approval are the DREAMM-7 (NCT04246047) and DREAMM-8 (NCT04484623) studies. FDA reviewers said the majority of patients in these trials experienced keratopathy and Visual Acuity (KVA) events. All grade KVAs occurred in 92% and 93% of patients in DREAMM-7 and DREAMM-8, respectively, while more serious grade 3-4 events were found in 77% and 78% of patients in the same two studies. GSK is looking to get Blenrep FDA-approved in combination with Takeda's Velcade and dexamethasone to treat adults with multiple myeloma who have already tried one prior line of therapy. The drugmaker is also seeking a positive decision for the antibody-drug conjugate (ADC) used in combo with Bristol Myers Squibb's Pomalyst and dexamethasone in the same proposed patient group. Blenrep originally won FDA approval in 2020 to treat multiple myeloma. Although initially getting ahead in the now lucrative ADC market, GSK pulled the drug in 2022 after confirmatory trial shortcomings. Since then, GSK has been busy conducting trials to showcase Blenrep's effectiveness in treating the rare type of blood cancer. In its own briefing document, the British big pharma company said that results from DREAMM-7 and DREAMM-8 'consistently demonstrated meaningful benefit across endpoints and supported the positive benefit-risk of Blenrep'. Data from the studies were enough to convince the European equivalent of the ODAC – the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) issued a positive recommendation for Blenrep in May 2025. However, the European Commission (EC) has not yet decided whether to approve the drug. The UK was the first country to authorise Blenrep in the revised treatment setting in April 2025. Buoyed by momentum in the wider ADC market, GSK has forecast more than £3bn in peak annual sales for Blenrep. Estimates by GlobalData – which go as far as 2031 – predict peak sales of $1.9bn. How ODAC votes will be a key factor in whether this sales figure is reached, however. While other ADCs are known for their side effects, a snag for GSK is that Blenrep seems to be the sole product in which eye safety has been a concern. "GSK's Blenrep US comeback hindered by eye safety concerns" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Insider
14-06-2025
- Business
- Business Insider
UroGen Pharma price target raised to $16 from $3 at Goldman Sachs
Goldman Sachs analyst Paul Choi raised the firm's price target on UroGen Pharma (URGN) to $16 from $3 and keeps a Neutral rating on the shares following yesterday's approval of Zusduri. The firm cites the stock being bid up (+22% following FDA approval of Zusduri in patients with low-grade intermediate-risk non muscle-invasive bladder cancer, which was surprising to most investor expectations given the negative ODAC advisory committee briefing document and vote that the drug's overall benefit-risk profile was not favorable, the analyst tells investors in a research note. Although the FDA is not obligated to follow the ODAC's recommendation, approvals following negative advisory committee meeting votes, particularly in oncology, are exceedingly rare, the firm adds. Confident Investing Starts Here:
Medscape
23-05-2025
- Health
- Medscape
FDA Questions Single-Arm Studies for Cancer Approvals
The US Food and Drug Administration's (FDA's) approval decision about a new treatment for non–muscle invasive bladder cancer (NMIBC) hinges on whether a single-armed trial can be trusted to adequately demonstrate risks and benefits. The issue came to a head May 21 when the agency asked its Oncologic Drugs Advisory Committee (ODAC) to weigh in on UGN-102 (UroGen Pharma), an intravesical mitomycin formulation meant to be an alternative to transurethral resection of bladder tumor (TURBT) for recurrent low-grade intermediate-risk NMIBC. UroGen presented results from its ENVISION trial in 223 patients with recurrent lesions on day 2 of a meeting of the FDA advisory committee. After 6 weekly 75 mg instillations, the complete response rate at the 3-month checkup was 77.6%. Twelve months after a complete response, almost 80% of subjects remained recurrence-free; at 18 months, just over half were still recurrence-free. UroGen highlighted the convenience of UGN-102, which can be instilled in a urologist's office, over TURBT, which is a surgical procedure usually done under general anesthesia. The company said UGN-102 'can reduce the burden of repeated TURBTs' in elderly, comorbid patients. However, there was no control arm in ENVISION, so outcomes vs TURBT, the current standard of care, are unclear. 'The lack of a concurrent control in the single-arm ENVISION trial makes interpretation of efficacy challenging,' the FDA said in a meeting document. Although the complete response rate indicates drug activity, the agency said the durations of response found in the study could simply be due to the fact that some patients with NMBIC recur, while others do not. The same holds true for safety. Most of the adverse events in the trial were genitourinary and low grade, but it's unclear if there are fewer than with TURBT. For one, patients were at risk for adverse events throughout the entire 6-week treatment window, whereas the duration of side effects with TURBT, as a single procedure, is generally shorter. 'The applicant has not demonstrated that treatment with UGN-102 is safer or more tolerable than TURBT,' the FDA said in the document. The FDA wanted UroGen to run a head-to-head randomized trial against TURBT. UroGen started one, but there was disagreement with the agency about how it should be conducted, and the trial ended early. How Committee Members Voted on Benefits vs Risks for UGN-102 On day 2 of the ODAC meeting, the FDA said the preliminary findings can't be used to compare the two approaches due to methodological issues. Richard Pazdur, MD, head of FDA's Oncology Center of Excellence, said there are also larger issues at play. If the agency approves UGN-102 for such a condition as common as NMIBC based on a single-arm study, 'guess what? Nobody's going to do a randomized trial. It goes to the lowest denominator for companies,' Pazdur said. The FDA didn't ask ODAC's opinion on approving UGN-102. Instead, it simply wanted committee members to vote on whether the overall benefit-risk of UGN-102 is favorable for recurrent, low-grade, intermediate-risk NMIBC based on the data. Five panelists voted 'no,' and four voted 'yes.' Radiation oncologist Daniel Spratt, MD, a prostate cancer specialist at Case Western Reserve University, Cleveland, was one of the 'no' votes. Without the randomized trial, he said, 'It's very hard to determine the true benefit of this, and there is toxicity.' Plus, 'this is a $140,000 treatment that might not change how many follow-up TURBTs you need after 3 months.' Another 'no' vote, Heidi McKean, MD, a community oncologist in Sioux Falls, South Dakota, said that 'in this population, a randomized control trial is feasible and would have really helped us understand the clinical meaningfulness of the intervention.' Surgical oncologist Mark Ball, MD, a kidney cancer specialist at the National Cancer Institute in Bethesda, Maryland, voted 'yes' in favor of UGN-102. The complete response rate at 12 months and beyond, 'even though there are differences in the interpretation of the data, is quite encouraging. I don't find the toxicity profile alarming, and therefore,' Ball said, 'the benefit-risk ratio is favorable.' Isla Garraway, MD, PhD, a urologic surgical oncologist at the University of California, Los Angeles, also voted 'yes,' saying it would be ideal to have a less invasive, in-office alternative to TURBT for the older patients most often affected by NMIBC. In the end, Pazdur said, 'This a relatively split vote, so we will be meeting with the sponsor to look at future directions.' ODAC was also asked for its thoughts on whether the FDA should require randomized trials for future low-grade, intermediate-risk NMIBC approval submissions. Committee members generally agreed, as long as the requirement doesn't delay the approval of promising treatments.
