Drug Discovery World

APRIL 3, 2023

This year, the team will launch a combined Phase I and IIa trial of R805/CX-011 for the treatment of osteoarthritis in patients in collaboration with the start-up Carthronix. The researchers genetically modified a mouse, substituting one amino acid for another in a way that prevents a specific GP130 signaling cascade from being activated.

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XTalks

DECEMBER 28, 2023

BillionToOne, a company specializing in molecular diagnostics, has partnered with Janssen Research & Development, LLC, a Johnson & Johnson division, to conduct the AZALEA Phase III clinical trial globally. The trial is set to occur across the US, the European Union (EU) and selected global locations.

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XTalks

SEPTEMBER 14, 2022

In this episode, Ayesha discussed the FDA approval of Sanofi’s enzyme replacement therapy Xenpozyme for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD), a rare genetic lysosomal storage disease, in adults and pediatric patients.

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Pharmaceutical Technology

MAY 3, 2023

PharmaTher has submitted a fast track application for Ketarx (ketamine) to the US Food and Drug Administration (FDA) to treat levodopa-induced dyskinesia in Parkinson’s disease (LID-PD). PharmaTher is now assessing the design of the Phase III trial to align with the recommendations of the FDA.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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XTalks

JANUARY 26, 2023

The US Food and Drug Administration (FDA) recently granted approval for the Bruton tyrosine kinase inhibitor (BTKi) Brukinsa (zanubrutinib) based on the results from two Phase III clinical trials. Brukinsa is now approved to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).

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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.” No adverse events were reported during the clinical trials following the administration of Adzynma. Adzynma’s approval is the second for Takeda this week.

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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

MARCH 10, 2023

This US Food and Drug Administration (FDA)-approved drug was also awarded a rare pediatric disease priority review voucher. Omaveloxolone Clinical Trials Skyclarys is an oral medication that contains omaveloxolone, an activator of the nuclear factor-erythroid 2 related factor 2 (Nrf2) pathway.

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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 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. XTALKS WEBINAR: All Means All: The Road to Inclusivity in Clinical Trials. Clinical Trials of Vtama. Dermavant’s Future Targets.

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Drug Discovery World

JANUARY 23, 2023

Armed with this understanding, the researchers further demonstrated that the genetic inhibition of DGAT1 suppresses radioresistance. In addition, they discovered that cladribine, a clinical drug, activates DGKB and inhibits DGAT1.

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Pharmaceutical Technology

AUGUST 16, 2022

According to the deal, Horizon will provide the funding to develop the therapy until the conclusion of its two Phase II clinical trials. Under the agreement, Horizon will hold an option for the acquisition of the ADX-914 programme, which is exercisable through a period after concluding the Phase II trials.

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Advarra

JUNE 20, 2023

of new cancer drugs tested in Phase I were likely to receive Food and Drug Administration (FDA) approval. Typical clinical development timelines for anticancer drugs average an estimated 6.7 Innovation Organizations conducting oncology clinical trials face challenges distinct from the rest of the research community.

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Trials Clinical Trials Clinical Trials FDA Approval 52

XTalks

APRIL 5, 2024

Amylyx Pharmaceuticals announced yesterday that it will be pulling its ALS (amyotrophic lateral sclerosis) drug Relyvrio from the market after failing in a confirmatory trial. Last month, Amylyx reported results from the global PHOENIX Phase III clinical trial that were unable to confirm the drug’s effectiveness.

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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.

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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). How Do Pombiliti and Opfolda Work?

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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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The Pharma Data

NOVEMBER 27, 2020

FDA Approves Imcivree (setmelanotide) for Chronic Weight Management in Patients with Obesity Due to POMC, PCSK1 or LEPR Deficiency. Nasdaq:RYTM), a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic diseases of obesity, announced today that the U.S. BOSTON, Nov.

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XTalks

MAY 17, 2024

The therapy is the first and only US Food and Drug Administration (FDA)-approved treatment that reduces the loss of kidney function in adults with the disease. He also traced his personal journey from the clinic to leading a rare disease biotech and winning FDA approval for a rare disease drug. Can they do it?

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Pharmaceutical Technology

JULY 29, 2022

CAMP4’s CSO David Bumcrot PhD tells Pharmaceutical Technology that the company plans to see clinical trials go forward for their urea cycle disorder programs late next year. However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea.

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XTalks

SEPTEMBER 17, 2021

The ALS Association is pushing for rapid FDA approval of Amylyx Pharmaceuticals Inc.’s After asking for a placebo-controlled Phase III study for the drug in April that upset the ALS Association, the FDA dialed back on the request this week, agreeing to accept only Phase II trial data for evaluation of Amylyx’s drug application.

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The Pharma Data

AUGUST 10, 2020

Genentech’s Evrysdi (risdiplam) has secured FDA approval for the treatment of spinal muscular atrophy (SMA), making it the first oral therapy available for the rare hereditary genetic condition in US and the second overall to be approved for the disease.

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FDA Approval Clinical Trials Clinical Trials Genetics 52

Delveinsight

AUGUST 17, 2021

FDA’s Green Flag to Keytruda and Lenvima combination by Merck and Eisai for Advanced renal cell carcinoma (RCC). The FDA approved the combination of Keytruda and Lenvima produced by Merck and Eisai, as a first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The rarity of disease – 1.36

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pharmaphorum

AUGUST 13, 2020

Viltepso can be used to treat around 8% of DMD patients and previously approved DMD drugs has not been shown to change the course of the disease. It’s bad form to compare performance of drugs in separate clinical trials, but results suggest that Viltepso helps patients produce more dystrophin than Sarepta’s rival Vyondys 53 (golodirsen).

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Drug Discovery World

OCTOBER 18, 2023

We’re beginning to see their potential come to fruition, with the FDA having approved three treatments as of January 2023 — for retinal dystrophy 1 spinal muscular atrophy, 2 and haemophilia B.

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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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pharmaphorum

AUGUST 18, 2022

.” The gene therapy specialist’s chief executive Andrew Obenshain said the approval “marks a watershed moment for the field of gene therapy” and is “ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”

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XTalks

FEBRUARY 28, 2022

Below are some facts and information about rare diseases, including rare disease clinical trials and orphan drugs. 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.

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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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Scienmag

DECEMBER 17, 2020

Credit: Stand Up To Cancer New York –December 16, 2020 — The FDA recently approved the immunotherapy drug pembrolizumab as a first-line treatment for patients with certain types of advanced colorectal cancer. This is the 9th FDA approval supported by Stand […].

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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. Outcomes of Clinical Trials for Breyanzi.

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Pharmaceutical Technology

MARCH 1, 2023

While the pandemic dramatically increased public awareness of mRNA therapeutics, lesser-known categories of RNA-based therapies have been established and approved for use by the FDA for almost a decade. These therapies validated antisense technology as a new platform for drug discovery and for targeting rare genetic disorders.

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WCG Clinical

MAY 28, 2024

CAR-T Cells Target Harmful B Cells in Lupus CAR-T cell technology, which uses genetic engineering to direct white blood cells to attack specific molecular targets, was originally proposed for treatment of HIV infection and hematological malignancies. The currently approved CD-19 CAR products are autologous, ex vivo cellular therapies.

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pharmaphorum

SEPTEMBER 16, 2022

With approximately one third of all current FDA-approved drugs targeting GPCRs, Verily/Sosei Heptares are looking to expedite GPCR research within not only immunology, but also gastroenterology and immuno-oncology as well, and the latest data bodes well for future development of therapeutic options in these areas. AI relies on data.

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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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Advarra

JANUARY 11, 2024

Adoptive T Cell therapies, therapeutic antibodies, and immunomodulatory proteins represent just some of the potentially beneficial treatment strategies for successful mRNA cancer trials. They must also seek institutional review board (IRB) approval, and often, oversight by an institutional biosafety committee (IBC) is also required.

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