The Pharma Data

AUGUST 9, 2021

Food and Drug Administration approved Nexviazyme (avalglucosidase alfa-ngpt) for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. “Pompe disease is a rare genetic disease that causes premature death and has a debilitating effect on people’s lives,” said Janet Maynard, M.D.,

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Conversations in Drug Development Trends

NOVEMBER 27, 2023

Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow. In June 2023, the U.S.

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BioPharma Drive: Drug Pricing

JUNE 11, 2024

The medicine will join Intercept’s Ocaliva as a treatment option for primary biliary cholangitis. Another drug, from the now Gilead-owned CymaBay, could be cleared for the condition by August.

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FDA Law Blog: Drug Discovery

DECEMBER 13, 2023

Sasinowski — On December 12, 2023, FDA announced the creation of a new advisory committee specifically for treatments for genetic metabolic diseases, the Genetic Metabolic Diseases Advisory Committee, or “GeMDAC.” Note that FDA is currently soliciting applications to staff this committee. Raver & Frank J.

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BioPharma Drive: Drug Pricing

OCTOBER 23, 2023

since late last year as the FDA sought more details on Verve’s in vivo treatment for heart disease. The trial, which is ongoing in the U.K. and New Zealand, has been on hold in the U.S.

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FDA Law Blog: Biosimilars

OCTOBER 14, 2024

California Stem Cell Treatment Center, Inc., At the clinics, Defendants offer treatments that consist of extracting fat tissue from patients and, through a multi-step process, turning that tissue into a liquefied mixture of stem cells, other cells and cell debris known as Stromal Vascular Fraction (SVF). By Steven J. 21 U.S.C. §

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The Pharma Data

OCTOBER 26, 2020

Food and Drug Administration (FDA) has approved EYSUVIS for the short-term treatment of dry eye disease. . adults have been diagnosed with dry eye disease, a chronic, episodic, multifactorial disease. EYSUVIS is the first FDA-approved corticosteroid specifically for dry eye disease treatment.

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Drug Target Review

OCTOBER 16, 2023

As we explore translatability, technological advancements and the integration of artificial intelligence (AI), we envision the potential role of PDOs throughout the entire medicine creation pipeline, from target identification to personalised clinical treatments, ultimately reducing costs and improving patient outcomes.

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The Pharma Data

NOVEMBER 12, 2021

Treatment is First FDA-Approved Option Patients Can Take Regardless of Previous Therapies. Food and Drug Administration approved Besremi (ropeginterferon alfa-2b-njft) injection to treat adults with polycythemia vera, a blood disease that causes the overproduction of red blood cells. The FDA, an agency within theU.S.

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Conversations in Drug Development Trends

OCTOBER 29, 2024

By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. Why Is There an Increase in Patient-Led Rare Disease Research?

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PLOS: DNA Science

OCTOBER 17, 2024

FDA approved a treatment for type 3 of the ultra-rare genetic disease September 20. The quest has been ongoing for three decades, said Laurie Turner, Family Services Manager of the National Niemann-Pick Disease Foundation. The new drug is taken with miglustat , which FDA approved for use in NPD3 in 2009.

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Conversations in Drug Development Trends

NOVEMBER 5, 2024

In preparation for World Orphan Drug Congress Europe, we interviewed Nathan Chadwick, Senior Director, Therapeutic Strategy Lead, Rare Disease, and Derek Ansel, MS, LCGC, Vice President, Therapeutic Strategy Lead, Rare Disease, to hear their insights into the current progress in rare disease research and their hopes for 2025.

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Drugs.com

SEPTEMBER 18, 2024

18, 2024 -- Women with early stage breast cancer may now take Kisquali, a medication already approved for advanced disease, following the U.S. Food and Drug Administration's expanded approval of the treatment, drug maker Novartis. WEDNESDAY, Sept.

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Drug Target Review

MARCH 12, 2024

The study’s findings offer new insights into the biochemical language microbes use to influence distant organ systems, which could revolutionise the way researchers approach disease. For example, bile acid treatments have been effective against SARS-Cov-2 infections. Bile acids and bile activated receptors in the treatment of Covid-19.

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Agency IQ

APRIL 19, 2024

FDA advisors endorse minimal residual disease (MRD) as accelerated approval endpoint for multiple myeloma Last week, FDA’s Oncologic Drugs Advisory Committee (ODAC) voted unanimously in favor of using minimal residual disease (MRD) as an accelerated approval endpoint for multiple myeloma.

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The Pharma Data

AUGUST 15, 2021

Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801, Vivet’s clinical-stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.

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FDA Law Blog: Drug Discovery

JUNE 12, 2024

Sasinowski — Hyman, Phelps & McNamara (HPM) would like to congratulate the 7 rare disease programs selected for the inaugural class of the FDA’s “Support for clinical Trials Advancing Rare disease Treatment” (START) pilot program. HPM is proud to aid 5 of the 7 selected participants for the START pilot program.

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BioPharma Drive: Drug Pricing

JULY 27, 2023

While the FDA doesn’t think the brain disease treatment merits an evaluation, Biohaven is still pushing for one and has requested a further meeting.

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PLOS: DNA Science

OCTOBER 19, 2023

Also recently, FDA’s Cellular, Tissue, and Gene Therapies Advisory Committe turned down a stem cell treatment for amyotrophic lateral sclerosis, aka ALS, Lou Gehrig’s disease, or motor neuron disease. ALS is a disease of adulthood, beginning typically between 40 and 70 years of age.

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The Pharma Data

AUGUST 7, 2021

Approval is based on positive Phase 3 data demonstrating improvements in key disease burden measures and establishing its safety profile Nexviazyme specifically targets the M6P receptor, the key pathway for enzyme replacement therapy, to effectively clear glycogen build-up in muscle cells. Targeted delivery to clear glycogen in muscle cells.

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The Pharma Data

APRIL 26, 2022

This action makes Veklury the first approved COVID-19 treatment for children less than 12 years of age. As COVID-19 can cause severe illness in children, some of whom do not currently have a vaccination option, there continues to be a need for safe and effective COVID-19 treatment options for this population,” said Patrizia Cavazzoni, M.D.,

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Conversations in Drug Development Trends

AUGUST 23, 2023

However, patients also play a vital role in engaging directly with the FDA. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA. He and McNary began working together several months later.

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BioPharma Drive: Drug Pricing

OCTOBER 18, 2023

Developed with Regeneron, Intellia’s treatment is designed to inactivate a gene to treat an inherited disease called transthyretin amyloidosis.

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Agency IQ

OCTOBER 6, 2023

In a first, FDA tackles treatments for stimulant use disorder A new draft guidance from the FDA gives developers a roadmap to advance the development of novel therapies to address stimulant use disorders. While stimulant use disorder is increasing, there are currently no FDA-approved medications.

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Agency IQ

JUNE 26, 2023

FDA quietly kicks off process that could inform future rare disease regulatory reforms Reforms buried within the FY2023 omnibus spending bill require the development of a new report looking at the regulatory processes of the U.S. Meanwhile, in the EU rare diseases are defined using a different set of criteria. In the U.S.,

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The Pharma Data

OCTOBER 8, 2021

Food and Drug Administration approved Rethymic for the treatment of pediatric patients with congenital athymia, a rare immune disorder. “Today’s action marks the first FDA approval of a therapy to treat this very rare and devastating disease in children,” said Peter Marks, M.D., Dosing is patient?customized,

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Drug Target Review

SEPTEMBER 19, 2024

Based on Nobel Prize-winning research, IRLAB has grown rapidly to become recognised and respected as a world-leader in understanding the complex neuropharmacology of CNS disorders, especially Parkinson’s disease (PD). The pipeline currently includes five drug candidates, all of which have the potential to revolutionise the treatment of PD.

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Eye on FDA

AUGUST 27, 2023

There are still many decisions and publicly available PDUFA dates with many novel treatments among them, including investigative treatments for sickle cell disease and ALS. During the first half of 2022, FDA issued only six press releases about drug approvals compared to 13 during the first half of this year.

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The Pharma Data

OCTOBER 18, 2021

The FDA’s Congressionally-funded Orphan Products Grants Program awards these grants to clinical investigators to support the development of medical products for patients with rare diseases. “These grants demonstrate the FDA’s commitment to supporting the development of new treatments for patients living with rare diseases.”

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Agency IQ

MARCH 15, 2024

BY AMANDA CONTI Given the progressive nature of Alzheimer’s disease (AD), developing treatments for early-stage populations is crucial. FDA has now issued a second revision to its draft guidance on developing drugs for this population. Fill out the form to read the full article.

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The Pharma Data

JULY 12, 2021

Food and Drug Administration (FDA) has approved finerenone, the first non-steroidal, selective mineralocorticoid receptor (MR) antagonist, under the brand name Kerendia ®. Chronic kidney disease (CKD) is a common and potentially deadly condition that is generally underrecognized. Bayer announced today that the U.S.

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Agency IQ

MAY 17, 2024

While most of the legislation being considered is not relevant to the FDA, four of the bills are. The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. A “markup” process involves votes on individual pieces of legislation, as well as amendments to those bills.

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The Pharma Data

OCTOBER 9, 2021

Food and Drug Administration (FDA) Antimicrobial Drugs Advisory Committee (AMDAC) voted unanimously to recommend use of maribavir (TAK-620) for the treatment of refractory cytomegalovirus (CMV) infection and disease with genotypic resistance to ganciclovir, valganciclovir, foscarnet or cidofovir in transplant recipients.

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BioPharma Drive: Drug Pricing

JULY 31, 2023

According to the company, FDA officials have signaled openness to using a surrogate endpoint for assessing accelerated approval of a treatment for limb-girdle muscular dystrophy.

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FDA Law Blog: Biosimilars

JANUARY 18, 2024

Houck — In August 2023 the Food and Drug Administration (“FDA”) and Health and Human Services (“HHS”) recommended that the Drug Enforcement Administration (“DEA”) reschedule marijuana from schedule I under the federal Controlled Substances Act (“CSA”) to schedule III. By Larry K. 53,688 (Aug. 53,767 (Aug.

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DrugBank

JULY 20, 2023

of the global population) suffer from rare conditions without effective treatments or cures. Rare diseases, therefore, present compelling opportunities for Drug Development. Since each disease is present in small groups of patients, this research is also fraught with complex challenges. At any given time, millions of people ( 3.5–5.9%

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The Pharma Data

OCTOBER 27, 2020

Food and Drug Administration (FDA) has granted Orphan Drug and Rare Pediatric Disease (RPD) designations to PBKR03 for the treatment of Krabbe disease (Globoid Cell Leukodystrophy). About Krabbe Disease. Passage Bio expects to initiate a Phase 1/2 trial for PBKR03 in the first half of 2021.

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