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

JANUARY 3, 2024

Wainua is the only FDA-approved drug for the treatment of ATTRv-PN that can be self-administered via an auto-injector. Approval of Wainua represents a meaningful advancement in treatment, one that gives those who are living with transthyretin-mediated amyloid polyneuropathy help managing the disease,” said Michael J.

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XTalks

DECEMBER 22, 2021

If the past year is anything to go by, then 2022 will also be a year marked by continued innovations in the life sciences. Here is a look at some life science trends to expect in the coming year, including key themes and topics in the pharmaceutical, biotechnology and medical device industries to keep an eye out for in 2022.

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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

OCTOBER 16, 2024

Hympavzi’s approval, with its innovative approach to balancing clotting, could offer a modern solution that adds to the global effort to reduce the risk of thrombosis. This is Pfizer’s second FDA-approved treatment for a rare genetic blood disorder this year.

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XTalks

APRIL 3, 2024

Related: Novartis’ Fabhalta Gets FDA Approval for Rare Complement Blood Disorder The FDA’s green light for Voydeya comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator in the world to back the therapy. AstraZeneca is also venturing into cell therapy and genetic medicine.

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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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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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XTalks

JUNE 23, 2022

In this episode, Ayesha and the team talked about the FDA approval of Eli Lilly’s JAK inhibitor Olumiant for the treatment of alopecia areata, also commonly just known as alopecia. The drug has become the first approved systemic treatment for the autoimmune disorder that causes patchy hair loss.

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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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Pharmaceutical Technology

FEBRUARY 13, 2023

Gene therapies are an attractive way of targeting the underlying genetic mutations, but traditional approaches cannot be used while targeting mutations for Dravet Syndrome, forcing researchers to develop new ways. SK Life Science is a subsidiary of SK, a South Korean holding company.

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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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XTalks

DECEMBER 19, 2024

Ensartinibs approval, granted under the commercial name Ensacove, was awarded to Xcovery Holdings, a subsidiary of Chinas Betta Pharmaceuticals. ALK-positive NSCLC is a subset of lung cancer driven by genetic rearrangements in the ALK gene. ALK-positive NSCLC is estimated to account for about five percent of all NSCLC cases.

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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

DECEMBER 18, 2024

The therapy is approved to be used in combination with glucocorticoid replacement therapies (steroids) to control androgen levels in adults and pediatric patients four years of age and older with classic CAH.

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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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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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XTalks

NOVEMBER 26, 2024

BridgeBio has been highly active in the genetic diseases space. Its investigational therapy infigratinib recently received Breakthrough Therapy designation, becoming the first-ever potential treatment for achondroplasia — a genetic condition affecting bone growth — to achieve this milestone.

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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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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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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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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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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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XTalks

MAY 15, 2023

Chiesi Global Rare Diseases in collaboration with Protalix BioTherapeutics announced the US Food and Drug Administration (FDA) approval of their new medication Elfabrio (pegunigalsidase alfa-iwxj) for the treatment of Fabry disease. Genetic confirmatory testing is then conducted to identify the specific mutations in the GLA gene.

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XTalks

SEPTEMBER 18, 2024

The US Food and Drug Administration (FDA) approved Kisunla (donanemab-azbt) , a treatment for patients with mild cognitive impairment or mild dementia caused by Alzheimer’s. To mitigate this, genetic testing for APOE4 is recommended before treatment.

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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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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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XTalks

JUNE 5, 2023

The genetic material of RSV (respiratory syncytial virus) encodes 11 proteins. Today’s FDA approval of Abrysvo recognizes significant scientific progress, and importantly helps provide older adults potential protection against RSV and an opportunity to improve community health by helping prevent the disease,” said Edward E.

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XTalks

DECEMBER 9, 2024

Programs like the Rare Disease Endpoint Advancement Pilot encourage collaboration with the FDA to establish novel surrogate endpoints. This initiative is particularly impactful for rare diseases and genetic disorders, where traditional endpoints may be infeasible.

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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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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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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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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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pharmaphorum

SEPTEMBER 12, 2022

Everyone’s genetic makeup differs, and each person’s cancer experience is unique to them – how cancer develops, how fast it spreads, which drugs it responds to, and more. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. Tailored oncology. About the author.

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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

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

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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