XTalks

JANUARY 2, 2024

For instance, Vyjuvek , the first FDA-approved gene therapy for DEB, is priced at $24,250 per vial. a biotech company specializing in the development and commercialization of genetic medicines for rare diseases, announced FDA approval for Vyjuvek for the treatment of DEB.

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XTalks

JUNE 20, 2022

Amvuttra’s FDA approval is based on positive data from the global, randomized, open-label, multicenter HELIOS-A Phase III study from a period of nine months. The condition has very limited treatment options. Amvuttra is administered via subcutaneous injection four times a year or once every three months (quarterly).

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XTalks

DECEMBER 29, 2022

The life sciences and healthcare are among the biggest industries globally, and their significance was particularly highlighted during the past couple of years by the COVID-19 pandemic. Given the hyperfocus on the life sciences thanks to COVID, consumers appear to be more autonomous and vocal about their medical demands and choices.

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XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Diagnosis is confirmed by a differential complete blood count and genetic testing. Treatment selection is determined by age, disease severity, comorbidities and the presence or absence of certain genetic mutations.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

SEPTEMBER 12, 2022

After approvals in Japan and Europe, Sanofi’s Xenpozyme has secured one from the US Food and Drug Administration (FDA) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Patients in the trial received Xenpozyme or placebo for 52 weeks.

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FDA Approval Genetics Trials Regulation 98

XTalks

DECEMBER 13, 2023

This approval also marks bluebird’s third ex vivo gene therapy approved by the FDA for a rare genetic disease and second FDA approval for an inherited hemoglobin disorder, cementing our position as a gene therapy leader.”

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Gene Therapy FDA Approval Gene In-Vivo 98

XTalks

NOVEMBER 10, 2023

Today, we are proud to further support the rare disease community by delivering Adzynma as the first FDA-approved therapeutic option for people with cTTP.” In 2022, the FDA approved 20 orphan drugs out of the 37 novel drugs approved by the FDA’s Center for Drug Evaluation and Research (CDER).

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

APRIL 11, 2024

After backing from the US Food and Drug Administration’s (FDA) Oncologic Drugs Advisory Committee (ODAC) last month, Johnson and Johnson (J&J) and Bristol Myers Squibb (BMS) scored FDA approvals for the use of their B-cell maturation antigen (BCMA)-targeting CAR T-cell therapies as earlier line treatment options for multiple myeloma.

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FDA Approval Manufacturing Trials Clinical Trials 52

XTalks

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. In the MOXIe Part 2 trial, 82 patients with Friedreich’s ataxia (as confirmed by genetic testing) were randomized 1:1 to receive either 150 mg omaveloxolone or placebo.

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FDA Approval Engineer Clinical Trials Clinical Trials 52

XTalks

JANUARY 27, 2023

The website brings awareness for facial angiofibroma, an aspect of the rare genetic disease tuberous sclerosis complex (TSC). Hyftor became the first FDA-approved topical treatment for facial angiofibroma associated with tuberous sclerosis.

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Life Science Genetic Disease FDA Approval Marketing 98

World of DTC Marketing

JANUARY 24, 2022

A group of early-stage life science investors and biotech CEOs joined together with a prominent patient advocate to speak out against H.R.3, The panel was hosted by the life sciences venture capital coalition Incubate and held at the National Press Club. Only about $2.2 3, known as the Lower Drug Costs Now Act.

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XTalks

MARCH 21, 2023

It is estimated that Rett syndrome affects 6,000 to 9,000 patients in the US, with a diagnosed population of approximately 4,500 patients, according to a press release from Acadia announcing Daybue’s approval. However, in animal studies, trofinetide has been shown to increase dendritic branching and synaptic plasticity signals.

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FDA Approval Gene Therapy Gene Genetics 94

XTalks

JUNE 29, 2022

The new FDA approval gives Breyanzi (lisocabtagene maraleucel) the broadest patient eligibility of any CAR T-cell therapy in relapsed or refractory LBCL. Treatment with Breyanzi is a tailored method of treatment which involves the genetic reprogramming of T cells. Product logo and product image courtesy of Bristol Myers Squibb.

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FDA Approval Gene Therapy Antibody Gene 52

XTalks

MAY 31, 2022

The precise cause of psoriasis is unknown, but risk factors include genetics and a family history of the condition, heavy alcohol consumption, stress, smoking and dry or cold weather. It has been estimated that around eight million people in the US and 125 million people in the world have been affected by psoriasis.

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FDA Approval Clinical Trials Clinical Trials Trials 98

XTalks

JUNE 16, 2023

Foundation Medicine recently announced that the US Food and Drug Administration (FDA) approved FoundationOne Liquid CDx to serve as a companion diagnostic for identifying metastatic colorectal cancer patients with BRAF V600E alterations, suitable for treatment with Pfizer’s Braftovi (encorafenib) with cetuximab.

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FDA Approval Genome Genomics DNA 97

XTalks

DECEMBER 20, 2023

As we step into 2024, the life sciences continue to evolve at an unprecedented pace, driven by technological innovation, a deeper understanding of human biology and the application of new technologies in areas like drug development and health wearables. These companies are at various stages of research and clinical trials.

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XTalks

SEPTEMBER 12, 2023

Life science podcasts have emerged as an invaluable tool for building connections with audiences in the digital era. Furthermore, we’ll explore the unique advertising opportunities that this platform offers, positioning brands at the forefront of the life science industry. The result?

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Life Science Branding Clinical Trials Clinical Trials 105

XTalks

MAY 17, 2023

In 2022, the FDA approved four new gene therapies, and more than a handful are looking to get approval this year for diseases including Duchenne muscular dystrophy (DMD), sickle cell disease (SCD) and hemophilia A. Of the more than 10,000 rare diseases, more than 80 percent are caused by genetic defects.

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Gene Therapy Clinical Trials Clinical Trials Gene 98

XTalks

NOVEMBER 27, 2023

Amicus Therapeutics has recently received approval from the US Food and Drug Administration (FDA) for its drugs Pombiliti (cipaglucosidase alfa-atga) and Opfolda (miglustat) for adults living with late-onset Pompe disease (LOPD) who are not improving on their current enzyme replacement therapy (ERT).

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FDA Approval Clinical Trials Clinical Trials Trials 104

XTalks

FEBRUARY 27, 2023

Chiesi Global Rare Diseases recently announced in a press release that Lamzede (velmanase alfa-tycv) received US Food and Drug Administration (FDA) approval for the treatment of non-neurological manifestations of alpha-mannosidosis in both adult and pediatric patients.

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FDA Approval Life Science Containment Genetics 52

XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Gene Therapy Gene FDA Approval Dermatology 97

XTalks

MARCH 28, 2024

For CRC, treatment options that are targeting genetic mutations such as BRAF , KRAS and HER2 have been developed. Building Cancer Awareness and Empowering Métis Youth: Featuring Jordyn Playne, President, Métis Nation of Ontario Youth Council – Xtalks Life Science Podcast Ep.

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Gene Editing Life Science Clinical Trials Clinical Trials 59

Drug Discovery World

APRIL 27, 2023

The report notes that the focus on science and pharma throughout the Covid-19 pandemic has resulted in the emergence of new locations as life science hubs and name checks Boston, Massachusetts and San Francisco, California as examples of centres with heightened investment appeal in the life sciences 1.

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XTalks

NOVEMBER 30, 2023

New FDA-Approved Treatments For HIV HIV treatment involves the administration of combined antiretroviral therapy (ART) to effectively suppress the viral load, maintain or enhance immune function and reduce the risk of opportunistic infections and cancers commonly associated with HIV. aiming to end the HIV epidemic by 2030.

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FDA Approval Gene Therapy Clinical Trials Clinical Trials 111

XTalks

MAY 12, 2023

Rett syndrome is a rare genetic disorder that predominantly affects girls and leads to severe physical and cognitive impairments. In March, the US Food and Drug Administration (FDA) approved Daybue (trofinetide) , the first and only treatment for Rett syndrome. It affects around one in 10,000 girls born each year.

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FDA Approval Genetics Trials Drugs 97

XTalks

AUGUST 21, 2023

The FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Ipsen that can be used for subsequent drug applications that would not qualify for a priority review. The FDA approval of Sohonos is a breakthrough for the US FOP community,” said Howard Mayer, head of R&D at Ipsen, in the company’s news release.

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Drugs FDA Approval Genetics Clinical Trials 98

XTalks

FEBRUARY 9, 2024

Epilepsy has diverse etiologies, which are categorized into structural, genetic, infectious, metabolic, immune and unknown causes. Ztalmy marks the first FDA-approved treatment specifically designed for CDD and operates as a neuroactive steroid, acting as a positive allosteric modulator of the GABA A receptor.

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FDA Approval Clinical Trials Clinical Trials Genetics 64

XTalks

APRIL 21, 2023

.” Cerner Enviza is a healthcare company headquartered in New York that specializes in life sciences data analysis and research. John Snow Labs is an American AI and NLP leader providing software, models and data consulting services for healthcare and life sciences industries.

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Life Science Allergies Drugs FDA Approval 98

XTalks

JANUARY 26, 2024

By targeting and identifying specific genetic mutations, this technology could lead to the development of more precise diagnostic tools, enabling early intervention and treatment. By tailoring treatments based on an individual’s genetic makeup, it may allow for more effective and targeted therapies.

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XTalks

AUGUST 15, 2023

Approximately 10 to 15 percent of patients with mCRPC have alterations in the BRCA gene — this subset of patients is likely to have aggressive disease, poor outcomes and a shorter survival time, according to information in a press release from Johnson & Johnson announcing Akeega’s FDA approval.

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FDA Approval Gene Hormones Licensing 52

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. She focuses on news relating to the food industry and writes blogs on recruitment and HR in the life sciences.

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Life Science HR Clinical Trials Clinical Trials 105

XTalks

MAY 1, 2023

Last week, the US Food and Drug Administration (FDA) granted accelerated approval to Biogen’s Qalsody (tofersen) for the treatment of adult patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 ( SOD1 ) gene.

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FDA Approval Gene Genetics Protein 89

XTalks

DECEMBER 28, 2023

Utilizing BillionToOne’s unique molecular counting technology, Quantitative Counting Templates (QCTs), the test employs next-generation sequencing (NGS) to detect genetic variants linked to the relevant fetal antigens. Such precise detection is crucial for accurately pinpointing patients most susceptible to developing severe HDFN.

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Trials Clinical Trials Clinical Trials Antibody 97

XTalks

FEBRUARY 24, 2023

The US Food and Drug Administration (FDA) granted accelerated approval to Genentech’s Lunsumio (mosunetuzumab) for adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have undergone two or more lines of systemic therapy. New treatment options are critically needed for FL.

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FDA Approval Drugs Antibody Medicine 52

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.

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Gene Therapy Gene Protein FDA Approval 98

XTalks

FEBRUARY 28, 2022

Are Most Rare Diseases Genetic? While 72 percent of rare diseases are genetic, not all are. Rare genetic diseases can be caused by either somatic or germline genetic changes. In 2020, the FDA approved 32 orphan drugs. In the US, 25 to 30 million Americans, or one in ten, are living with a rare disease.

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Clinical Trials Clinical Trials Genetics Trials 98

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.

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FDA Approval Drugs Sales Development 101