XTalks

DECEMBER 28, 2023

XTALKS WEBINAR: Impacts of the New IVD Regulation (IVDR) for Manufacturers and Users Live and On-Demand: Tuesday, January 9, 2024, at 10am EST (4pm CET/EU-Central) Register for this free webinar to learn how the new In Vitro Diagnostic Regulation (IVDR) impacts manufacturers and users of diagnostic devices.

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Trials Clinical Trials Clinical Trials Antibody 97

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. The MeCP2 protein plays a crucial role in regulating the activity of genes involved in brain development. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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FDA Approval Genetics Trials Drugs 97

XTalks

JUNE 28, 2023

The approval is restricted to ambulatory patients within this narrow age range due to uncertainty around its effectiveness in older children, which Sarepta hopes to clarify in a confirmatory trial. DMD is a rare genetic disorder that leads to progressive muscle degeneration and weakness.

Gene Therapy 98

Gene Therapy Gene Protein FDA Approval 98

XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s Accelerated FDA Approval Modeled on Aduhelm. This has brought criticism that US regulators are moving too slow on a drug that has clinical promise and is being backed by other international regulators.

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Trials Drugs FDA Approval Clinical Trials 98

XTalks

MARCH 25, 2021

Xtalks is proud to announce the launch of the Xtalks Life Science podcast. Subscribe to the Xtalks Life Science Podcast to never miss a new episode. Fresh Conversations About Life Science Topics. Diverse Voices for Lively Discussions.

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Life Science HR Clinical Trials Clinical Trials 105

Pharmaceutical Technology

NOVEMBER 29, 2022

PharmaTher also has an agreement in place with Case Western Reserve University to develop ketamine as a treatment for Rett syndrome, a rare genetic neurological disorder. In 1970, the FDA approved ketamine as an anesthetic. The company now plans to move this approach into a Phase III trial.

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Research Trials Drugs Generic Drugs 275

XTalks

FEBRUARY 5, 2024

In the dynamic landscape of oncology therapeutics, understanding the trends and performances of leading drugs is crucial for stakeholders across the healthcare and life sciences sectors. The top 40 best-selling oncology drugs in 2023, informed by 2022 sales statistics, mark significant milestones in cancer treatment and research.

FDA Approval 101

FDA Approval Drugs Sales Development 101

XTalks

AUGUST 6, 2020

The US Food and Drug Administration (FDA) has approved the use of an oral cannabidiol (CBD) solution called Epidiolex for the treatment of seizures associated with tuberous sclerosis complex (TSC) in patients aged one year and older. Epidiolex is the only FDA-approved formulation that contains CBD derived from the cannabis plant.

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FDA Approval Protein Containment Genetic Disease 64

XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

Gene Therapy 95

Gene Therapy Gene Trials Clinical Trials 95

XTalks

FEBRUARY 5, 2024

In the dynamic landscape of oncology therapeutics, understanding the trends and performances of leading drugs is crucial for stakeholders across the healthcare and life sciences sectors. The top 40 best-selling oncology drugs in 2023, informed by 2022 sales statistics, mark significant milestones in cancer treatment and research.

FDA Approval 52

FDA Approval Drugs Sales Development 52

XTalks

NOVEMBER 7, 2022

Immunofluorescent staining of CTCs is a CTC isolation method that has been used since the first US Food and Drug Administration (FDA) approval in 2013 for the CellSearch ® CTC enumeration platform. Regulatory Approvals for CTC-Based Assays in Oncology. Table 1: Some examples of cfDNA-based assays that are approved by the US FDA.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

Cloudbyz

MAY 19, 2021

Introduction Cell and gene therapy is an upcoming wave of therapeutic innovation in the healthcare and life sciences industry and is being pragmatically accepted worldwide. The gene therapy market reported its first market approvals back in 2017 and the evolution has been extensive ever since.

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Gene Therapy Gene Manufacturing Production 52

Drug Discovery World

AUGUST 3, 2023

Medical cannabis is often a first-thought when regarding plant-based medicines, but there remains public scepticism, arguably due to the regulations relating to the substance. For example, the FDA has not approved a marketing application for cannabis for the treatment of any disease or condition to date 1.

Medicine 52

Medicine Scientist Production Regulation 52

XTalks

SEPTEMBER 17, 2020

Often, the potential patient pool for a given trial is small and the unmet need is high, incentivizing drugmakers and regulators to find innovative ways to make game-changing therapeutics available to those who need them most. Nizoral is prescribed off-label, while the others are FDA approved. Duchenne Muscular Dystrophy.

Trials 98

Trials Gene Hormones Clinical Trials 98

XTalks

NOVEMBER 20, 2023

The newly approved Truqap and Faslodex combo is restricted to patients with a genetic alteration in one or more of the following PI3K/AKT pathway genes: PIK3CA , AKT1 or PTEN. PI3K/AKT is a key intracellular signaling pathway that regulates cell survival and proliferation. It was also given FDA priority review.

HR 98

HR FDA Approval Gene Trials 98

XTalks

DECEMBER 12, 2023

Importantly, FDA-approved products are now available for these PET imaging applications. The approvals have unlocked the potential to use these highly sensitive agents to assess patient eligibility, determine target engagement and evaluate treatment efficacy for Alzheimer’s disease.

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Trials Clinical Trials Clinical Trials Genetics 111

XTalks

JULY 27, 2022

The exact causes of ADHD are unknown, but genetics may play a key role. XTALKS WEBINAR: Delivering Patient Data through a Regulated Platform to Improve Trial Oversight. RELATED: Qelbree: The First FDA-approved Nonstimulant ADHD Drug for Adults in 20 Years. The Benefits of a Long-Acting Treatment.

Clinical Trials 98

Clinical Trials Clinical Trials Trials FDA Approval 98

XTalks

FEBRUARY 27, 2024

There have been significant advancements in new rare disease drugs, particularly for genetic disorders that can be treated by correcting, replacing or silencing defective genes. In 2020, 31 out of 53 novel drug approvals were for rare or orphan diseases​​. The FDA introduced its Orphan Drug Act in 1983.

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Research Drugs Gene Therapy Clinical Trials 59

XTalks

MARCH 13, 2023

The US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) held a recent town hall where three experts from the regulator provided guidance on how to design and conduct gene therapy clinical trials for rare diseases. There are around 300 million people worldwide living with a rare disease.

Gene Therapy 52

Gene Therapy Clinical Trials Clinical Trials Gene 52

XTalks

APRIL 13, 2022

About 80 percent of rare diseases have a genetic basis. Dr. Hale gave an example of how a natural history study was used as a control that led to the FDA approval of alglucosidase alfa in Pompe disease, a pediatric metabolic disorder affecting the cardiac and skeletal muscle.

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Clinical Trials Clinical Trials Clinical Research Trials 98

Drug Discovery World

AUGUST 10, 2022

Finally, in 2017, after years of improving T cell engineering techniques, the first chimeric antigen receptor (CAR) T cell therapy received FDA approval. Since 2017, five CAR T cell therapies have been approved and have shown incredible success in the clinic. CAR T cells are T cells that have been trained to attack tumours.

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Manufacturing Gene Genome Genomics 98

XTalks

AUGUST 30, 2022

Nusinersen was the first FDA-approved treatment for spinal muscular atrophy (SMA), the first approved nucleic-acid therapeutic for a neurologic disease and the first approved drug to correct defective splicing. Direct CNS administration is also FDA-approved for multiple small molecules and ASOs.

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Trials Drug Delivery Drugs Gene 83

XTalks

APRIL 12, 2021

The gene therapy company was focused on applying adeno-associated virus (AAV) vectors to developing a one-time treatment for patients with Parkinson’s disease who have specific genetic mutations. But just three months later, Voyager faced another stumbling block in the form of an FDA-mandated clinical hold on the trial.

Gene Therapy 96

Gene Therapy Gene Trials Development 96

XTalks

DECEMBER 24, 2020

The pandemic propelled the life science and healthcare sectors onto center stage, and they rose to the occasion against the most unprecedented health challenge in recent times. While COVID-19 has undoubtedly been the biggest story in the life science industry in 2020, it was a busy and positive year in many other areas.

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Life Science Vaccination Vaccine Gene 111

Pharmaceutical Technology

JANUARY 22, 2023

In 2022, the FDA approved only 37 new medicines, an underwhelming number compared to 98 in 2018. However, while only around 34% of the approvals in 2018 were for orphan drugs, 54% new approvals in 2022 were for drugs to treat rare diseases. T3 is a major hormone in the blood that regulates thyroid levels.

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Drugs Gene Hormones Clinical Trials 317