pharmaphorum

MAY 13, 2021

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Regardless of cohort, eligible participants are scheduled to receive the investigational gene therapy, either at the start of the study or after one year following treatment with placebo.

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STAT News

NOVEMBER 23, 2022

Food and Drug Administration on Tuesday approved the first gene therapy to treat people with hemophilia B, an inherited bleeding disorder , STAT writes. And finally, no matter how full you get, there’s always room for dessert — and yes, for an extra helping of gratitude. Enjoy the holiday.

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Gene Therapy FDA Approval Gene Genetics 97

XTalks

OCTOBER 22, 2024

transmembrane tight junction protein, which is commonly found in some gastric and GEJ cancers. Vyloy is approved to be used in combination with chemotherapy (fluoropyrimidine- and platinum-based) as a first-line treatment for patients with locally advanced, unresectable or metastatic forms of these cancers.

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FDA Approval Drugs Licensing Licensing 105

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. The condition affects one in 10,000 to 15,000 live female births.

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Drugs Gene Hormones Clinical Trials 317

pharmaphorum

MAY 21, 2021

US biotech bluebird bio has had a challenging time in the last few months, so a recommendation for EU approval of its gene therapy for adrenoleukodystrophy (ALD) will give it a lift. The treatment restore the activity of a protein that breaks down the toxic metabolites.

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Gene Therapy Gene FDA Approval Clinical Trials 96

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). It has been modified so that it cannot replicate in normal cells.

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Gene Therapy Gene FDA Approval Dermatology 97

Pharmaceutical Technology

MAY 15, 2023

In a closely contested debate, a US Food and Drug Administration Advisory Committee (AdCom) panel voted eight to six in favour of the accelerated approval for Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) gene transfer therapy delandistrogene moxeparvovec (SRP-9001).

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Gene Gene Therapy FDA Approval Drugs 130

XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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Gene Therapy Gene FDA Approval Antibody 98

XTalks

JANUARY 26, 2023

This is the fourth US FDA approval that BeiGene has received for Brukinsa since 2019. Tyrosine Kinase Inhibitors for Cancer Treatment Tyrosine kinases, or TKs, are a group of signaling molecules that affect multiple downstream proteins.

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FDA Approval Life Science Clinical Trials Clinical Trials 97

XTalks

DECEMBER 27, 2022

Ferring Pharmaceuticals announced that the US Food and Drug Administration (FDA) approved Adstiladrin (nadofaragene firadenovec-vncg) for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.

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Gene Therapy FDA Approval Gene Clinical Trials 52

pharmaphorum

AUGUST 10, 2020

Roche is hoping to undercut hugely expensive rivals after the FDA approved its oral spinal muscular atrophy (SMA) drug Evrysdi (risdiplam). The FDA approved Evrysdi for the treatment of spinal muscular atrophy (SMA) in adults and children two months of age and older. Zolgensma costs about $2.1m

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FDA Approval Drugs Gene Therapy Protein 59

pharmaphorum

NOVEMBER 12, 2021

Researchers in the US have developed an artificial intelligence-based tool that is able to predict COVID-19 symptoms and suggest which FDA-approved drugs might be used to treat patients.

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Doctors Doctor Drugs Gene Expression 135

Pharmaceutical Technology

APRIL 18, 2023

This news follows the treatment’s FDA approval on 10 March, making it the first-approved Rett Syndrome treatment in the US. The FDA approved the drug based on positive Phase III data from the Lavender trial. We have worked hard to make Daybue available as quickly as possible following FDA approval.”

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Drugs FDA Approval Clinical Trials Clinical Trials 255

Pharmaceutical Technology

AUGUST 23, 2022

On obtaining the FDA EUA, the Omicron BA.4/BA.5-adapted It comprises messenger ribonucleic acid (mRNA) that encodes for the initial spike protein of SARS-CoV-2 along with mRNA that encodes for Omicron BA.4/BA.5 5 variant spike protein. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Vaccination Vaccine Gene Therapy Protein 130

XTalks

NOVEMBER 10, 2023

The US Food and Drug Administration (FDA) has approved Takeda Pharmaceuticals’ Adzynma, the first recombinant protein product for prophylactic (preventive) or on‑demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), an ultra-rare blood clotting disorder.

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FDA Approval Gene Therapy Gene Clinical Trials 59

XTalks

AUGUST 17, 2020

Duchenne muscular dystrophy (DMD) patients with a mutation in exon 53 of the dystrophin gene now have a second approved treatment option, and Xtalks spoke with Sharon Hesterlee, PhD, Muscular Dystrophy Association executive vice president and chief research officer, to learn more.

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Drugs Clinical Trials Clinical Trials Gene 105

XTalks

NOVEMBER 24, 2022

In a pivotal approval, the US Food and Drug Administration (FDA) has given the nod to a new gene therapy called Hemgenix (etranacogene dezaparvovecfor) for the treatment of adults with the genetic blood disorder hemophilia B (congenital Factor IX deficiency). With a list price of $3.5 Hemophilia B primarily affects men.

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Gene Therapy Gene FDA Approval Drugs 52

XTalks

NOVEMBER 30, 2022

In this episode, Ayesha shared news about the FDA approval of the first gene therapy for the rare blood disorder Hemophilia B. Read the full articles here: Hemgenix Approved as First Gene Therapy for Hemophilia B. World Antimicrobial Resistance (AMR) Awareness Week: Preventing AMR Together.

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Gene Therapy Life Science Gene FDA Approval 52

The Pharma Data

AUGUST 13, 2020

NS Pharma has claimed a tentative FDA approval for its Viltepso (viltolarsen) injection in the treatment of Duchenne muscular dystrophy (DMD) in patients who are suitable to receive exon 53 skipping therapy. Mutations in the DMD gene can lead to an absence of the muscle cell protein dystrophin.

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FDA Approval Genetics Protein Gene 52

pharmaphorum

OCTOBER 18, 2022

Eli Lilly has agreed to buy hearing loss gene therapy developer Akouos in a deal that could value the company at around $610 million – if its lead candidate progresses as hoped in the clinic. There are no approved drug treatments for this type of hearing loss. The buyout – for $12.50 The buyout – for $12.50

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Gene Therapy Gene Genetics FDA Approval 52

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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FDA Approval Gene Genetics Clinical Trials 98

XTalks

NOVEMBER 1, 2024

Why it sold so well: Botox is FDA-approved for the treatment of chronic migraines and that is currently the only chronic condition for which it has received approval. Related: Top 30 Drugs to Watch in 2024: Insights from 2023 Sales Data 1. billion in the US and $4.1 billion worldwide last year.

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FDA Approval Drugs Botox Sales 85

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. A gene therapy, it permits the body to read over the DNA mutation and still continue to produce dystrophin. The post Translarna not approved by NICE for DMD on NHS appeared first on.

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Gene Therapy Gene FDA Approval DNA 98

The Pharma Data

MAY 31, 2022

The approval is based on interim efficacy and safety data from the RAINBOWFISH study in newborns, which showed that the majority of pre-symptomatic babies treated with Evrysdi achieved key milestones such as sitting and standing with half walking after 12 months of treatment. All infants were alive at 12 months without permanent ventilation.

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FDA Approval Protein In-Vitro Medicine 52

pharmaphorum

SEPTEMBER 12, 2022

Cancer cells and tumours behave differently than normal cells and have changes in their genes that make them different from an individual’s normal cells. Furthermore, each cancer has various gene changes – also referred to as the genetic makeup of cancer. In other words, every person’s cancer has its own genetic construct.

Genome 105

Genome Genomics Clinical Trials Clinical Trials 105

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

MAY 12, 2023

Caused by mutations in the MECP2 gene located on the X chromosome, Rett syndrome is primarily an X-linked disorder; however, there have been rare cases of boys being affected, usually with more severe symptoms due to the absence of a second X chromosome. Daybue (trofinetide) is the first and only FDA-approved drug for Rett syndrome.

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FDA Approval Genetics Trials Drugs 97

XTalks

SEPTEMBER 25, 2024

In a significant regulatory milestone, the US Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for treating neurological symptoms associated with Niemann-Pick disease type C (NPC). NPC, caused by mutations in the NPC1 or NPC2 gene, typically shortens life expectancy to around 13 years.

FDA Approval 52

FDA Approval Drugs Protein Gene 52

XTalks

SEPTEMBER 17, 2020

Nizoral is prescribed off-label, while the others are FDA approved. Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria. Dietary changes, including restriction of salt and animal protein, are also recommended. galactosidase A (?-Gal

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Trials Gene Hormones Clinical Trials 98

Pharmaceutical Technology

MAY 23, 2023

The US Food and Drug Administration (FDA) has granted Centessa Pharmaceuticals’ activated Protein C (APC) inhibitor SerpinPC a fast track designation for treating haemophilia B. The FDA’s fast track designation is designed to speed up the regulatory process to allow faster drug development and review to fill unmet needs.

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Gene Therapy Gene FDA Approval Drugs 147

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

Find Me Cure

OCTOBER 13, 2019

HER2 is a protein that plays an important role in cancer cell growth and is found on the surface of cancer cells in a very high quantity in about 10-20% of breast cancers. There are antibody drugs targetting those cells that are already FDA approved and there are antibody drugs still in trials.

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Development DNA Clinical Trials Clinical Trials 52

XTalks

JUNE 9, 2022

When a child born from parents with FH genes, inherits one FH gene from each parent, they might develop homozygous familial hypercholesterolemia (HoFH). Evkeeza works by binding to and blocking the function of ANGPTL3, which is a protein that plays a role in lipid metabolism. This disease can also result in early fatalities.

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Trials Antibody Genetics Gene 52

The Pharma Data

JULY 6, 2021

Enrolment is currently underway for a pivotal Phase 3 trial for AL001 in people at risk for or with frontotemporal dementia due to a progranulin gene mutation (FTD-GRN). FTD-GRN is a rapidly progressing and severe form of dementia found most frequently in people less than 65 years old at the time of diagnosis and has no approved treatments.

Genetics 52

Genetics Gene Antibody Development 52

XTalks

MARCH 1, 2024

Related: Interview with Neuromod COO Diarmuid Flavin About the FDA Approval of Tinnitus Treatment Lenire – Xtalks Life Science Podcast Ep. Gene Therapies for Hearing Loss Gene therapies to correct genetic mutations that cause hearing loss is an active area of development.

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Gene Therapy Gene Life Science Genetics 52

XTalks

DECEMBER 12, 2023

Additionally, the potential for accelerated approval of therapies via biomarker surrogate endpoints is introducing new complexities into the mix. “In This approval was based on the observed reduction of plasma neurofilament light chain (NfL) in patients who received tofersen.

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Trials Clinical Trials Clinical Trials Genetics 111

XTalks

AUGUST 30, 2022

We know that a lot of different pathologies, especially as we’ve entered the era of molecular medicine and gene therapies, cause many of the same symptoms and work through final common pathways. What About Small Proteins and Antibodies Reaching Extracellular CNS Targets? Viral Vectors for Gene Delivery to the CNS.

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Trials Drug Delivery Drugs Gene 83