FDA approval Archives - Medical Design Sourcing

How the FDA approval of J&J’s Opsynvi could simplify treatment and improve outcomes for PAH patients

[J&J]

Pulmonary arterial hypertension (PAH), a rare, progressive and life-threatening blood vessel disorder, affects some 500 to 1,000 new patients each year in the U.S. FDA recently approved Opsynvi, a first-of-its-kind once-daily single-tablet combination therapy from Johnson & Johnson.

“With this approval, our portfolio now includes treatments that address all three guideline-recommended pathways,” said a J&J spokesperson, highlighting the therapy’s alignment with current clinical guidelines recommending early use of combination therapy. The three treatment pathways include the endothelin, prostacyclin and nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) pathways.

The impact of Opsynvi PAH combination therapy

Before the approval, the DUE study for the combination therapy was featured in The Journal of the American College of Cardiology. The tria…

How the FDA approval of J&J’s Opsynvi could simplify treatment and improve outcomes for PAH patients

[J&J]

Pulmonary arterial hypertension (PAH), a rare, progressive and life-threatening blood vessel disorder, affects some 500 to 1,000 new patients each year in the U.S. FDA recently approved Opsynvi, a first-of-its-kind once-daily single-tablet combination therapy from Johnson & Johnson.

“With this approval, our portfolio now includes treatments that address all three guideline-recommended pathways,” said a J&J spokesperson, highlighting the therapy’s alignment with current clinical guidelines recommending early use of combination therapy. The three treatment pathways include the endothelin, prostacyclin and nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) pathways.

The impact of Opsynvi PAH combination therapy

Before the approval, the DUE study for the combination therapy was featured in The Journal of the American College of Cardiology. The tria…

Could Wegovy’s cardiovascular label expansion be a catalyst for GLP-1 obesity drug coverage?

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The recent FDA approval of a cardiovascular risk reduction indication for Wegovy (semaglutide) could point toward a significant opportunity for pharma companies seeking to reshape payer perceptions and expand coverage for next-gen metabolic therapies. This regulatory shift, allowing Wegovy to be prescribed for reducing the risk of major adverse cardiovascular events such as heart attack and stroke in addition to weight loss, aligns with mounting payer pressure to address the worsening obesity epidemic, which could impact 60% to 80% of the population of Western nations by the middle of the century. The dynamic has the potential to break down long-standing reimbursement barriers for effective yet costly medications.

Payers reckoning with the complex economics of obesity

There were already signs of change. While around 25% of employers provided coverage for GLP-1 drugs like Wegovy and Ozempic …

How Novo Nordisk’s Wegovy cardiovascular benefits could drive further growth

Semaglutide was already one of the best-selling drugs of recent memory. And Novo Nordisk the fastest-growing Big Pharma firm. Now, the FDA’s decision to expand the label of its weight-loss version of the drug to include cardiovascular benefits could help unlock more growth momentum for Novo Nordisk. This positions Wegovy as the first weight-loss medication also cleared for reducing the risk of heart attack, stroke, or heart-related death in at-risk individuals. The approval, based on a 17,600-patient SELECT study demonstrating a 20% lower risk of cardiac events with Wegovy versus placebo, could have a material impact on sales. The study also found that patients lost an average of 15% of their weight.

The expanded FDA label sanctions the use of Wegovy for reducing the risk of cardiovascular death, heart attack, and stroke in adults with cardiovascular disease who are obese or overweight. This marks a significa…

Could LSD change the game in anxiety treatment?

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A once-controversial psychedelic substance could potentially be a promising treatment for generalized anxiety disorder (GAD). That’s the view of Dr. Rakesh Jain, a psychiatrist with extensive experience in clinical practice, research, and education, affiliated with Texas Tech University School of Medicine. Jain expressed optimism in LSD-based therapy while acknowledging the challenges inherent in such a radical shift, in a recent MindMed analyst briefing on the heels of its MM-120 winning a breakthrough designation for an LSD-based treatment for GAD, based on promising topline data from a phase 2b study.

A once-controversial psychedelic substance could hold the key to treating Generalized Anxiety Disorder (GAD). This is the view of Dr. Rakesh Jain, a prominent psychiatrist who serves as a clinical professor at the Department of Psychiatry at Texas Tech University School of Medicine. While ack…

Eicos Sciences’ FDA-approved Aurlumyn promises to drastically reduce frostbite amputations

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Imagine scaling a mountain only to later lose digits or an entire hand to the unforgiving ravages of frostbite. For climbers, explorers, adventurous backcountry skiers, and even those caught in unexpected winter storms, severe cold exposure carries a devastating risk — amputation. Today, thanks to a potentially landmark FDA approval, the prospect of losing one’s extremities to frostbite becomes slightly less chilling.

Novel indication for iloprost

Eicos Sciences’s Aurlumyn, containing the active ingredient iloprost, offers a novel approach to treating severe frostbite. Iloprost, a synthetic version of a naturally occurring substance known as prostacyclin, targets two key problems. First, it addresses cold-induced blood vessel construction by widening them to restore essential blood flow to the affected tissues. Second, it prevents blood clots from forming, which can further block circulation…

A timeline of Aduhelm’s rise and fall

The story of Aduhelm has been rocky now for years. Biogen turned heads when its controversial Alzheimer’s therapy won accelerated approval from the FDA against the advice of its own advisory panel in mid-2021. The company had high hopes for the antibody at that point, pricing it at an average of $56,000 per year. Aduhelm would encounter a series of blows after that – rejected coverage, dismal sales, discontinued trials. Biogen would ultimately pull the plug on the drug this year.

Now, Biogen aims to prioritize other Alzheimer’s programs like the lecanemab, which was jointly developed with Eisai and FDA-approved in July 2023. Biogen noted in a statement that insights gained from Aduhelm hold value for the company. Ultimately, Biogen recorded a $60 million charge tied to wrapping up Aduhelm’s development.

A timeline showing the ups and downs in Aduhelm’s journey in reverse-chronological order follows. Stock data are from Yfinance and annotation chart …

Caribou Biosciences’ CEO discusses CRISPR progress, future goals, and gender equality in biotech

Founded in 2011, Caribou Biosciences is a pioneer in the development of CRISPR genome editing technologies, a field honored with the Nobel Prize in Chemistry in 2020. Co-founded by Jennifer Doudna, Ph.D., one of the Nobel laureates, and CEO Rachel Haurwitz, and two other CRISPR pioneers, the company has raised over $800 million in funding, including significant investments from industry giants like Pfizer. In 2021, the company entered the clinic with their lead program and completed a successful $350 million IPO.

“Today, we’re laser focused on using Caribou’s next generation CRISPR technology, which we call chRDNA technology to advance a pipeline of wholly owned off the shelf CAR-T and CAR-NK cell therapies,” said Haurwitz in a recent interview at the JP Morgan Health Care conference.

CRISPR: From lab to clinic in about a decade

Rachel Haurwitz, Ph.D., CEO of Caribou Biosciences

Rachel Haurwitz,…

Using AI to unlock new uses for existing cancer medicines

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Repurposing is a drug development strategy that has been widely applied in cancer. This strategy, sometimes called label expansion, involves obtaining FDA approval to market a drug for the treatment of new indications, alone or in combination with other drugs. Not only can this approach extend the window of patent protection for a commercialized drug, but the path to regulatory approval can also be far less costly and risky than the traditional approach to drug development, largely because clinical evidence of safety already exists. And most importantly, the result is more and better treatment options for patients.

In keeping the law of unintended consequences, recent provisions introduced by the 2022 Inflation Reduction Act (IRA) blunted the potential benefits of drug repurposing. As a result of limiting the window of time during which pharmaceutical companies can recoup investments before…

MDMA’s potential shift from party drug to PTSD therapy could hinge on strict safety measures

MDMA, the stimulant mood-lifting drug commonly known as ecstasy, could soon transition from party staple to FDA-approved medication — but likely with tight control measures to address its abuse potential and safety risks.

On Tuesday, the nonprofit Multidisciplinary Association for Psychedelic Studies (MAPS) filed an application seeking FDA approval of MDMA-assisted therapy for post-traumatic stress disorder, based on positive phase 3 results demonstrating significant PTSD reductions for most trial participants. In the studies, a significant number of those receiving MDMA-assisted therapy no longer qualified for a PTSD diagnosis after three treatment sessions, with a large majority seeing clinically meaningful improvements.

Currently, the Drug Enforcement Administration (DEA) categorizes MDMA as a Schedule I controlled substance having no approved medical uses, although it is likely to reclassify the stimulant if it receives FDA approval.

MAPS founder Ric…

Cybin sees near future for psychedelic therapy after promising interim phase 2 data

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With promising interim phase 2 data in hand, Cybin believes psychedelic therapy will become a reality in the “not too distant future,” according to CEO Doug Drysdale.

As recently as the 1990s, it would be difficult to imagine that a psychedelic drug would potentially be a clinical option for a mood disorder like depression. But before long, data from multiple phase 3 trials will be available on psilocybin derivatives for various mood disorders.

Interim phase 2 CYB003 data as a ray of hope for treating mood disorders

Toronto, Canada–based Cybin recently unveiled upbeat interim phase 2 data related to CYB003, a proprietary deuterated psilocybin analog, in patients with moderate to severe major depressive disorder (MDD). In the randomized double-blind placebo-controlled study, CYB003 demonstrated a rapid and robust reduction in depression symptoms just three weeks …

Lilly’s Zepbound to enter the weight management market with competitive pricing

Lilly’s tirzepatide notched an FDA approval for chronic weight management, potentially clearly the way for billions in additional sales. Analysts have projected that the drug could fetch $26 billion in annual sales by 2030, with roughly two-thirds of that sum related to obesity treatment. Bank of America analyst Geoff Meacham is even more optimistic, predicting that sales could hit $48 billion annually.

The company is calling the obesity-branded version of the drug Zepbound, in contrast to Mounjaro, which won FDA approval for type 2 diabetes on May 13, 2022.

Lily’s stock was up 3.20% to $619.13. Year-to-date, its share price is up almost 70%, in part as a result of strong sales prospects for tirzepatide.

The table below outlines the starting cash prices for a range of GLP-1 receptor agonists. Lilly’s Zepbound, the obesity-branded version of tirzepatide, has a competitive starting price in the segment. With insurance, Zepbound could cost as lo…