Advarra

JULY 26, 2022

The 2018 Farm Bill. The 2018 Farm Bill removed hemp from the Controlled Substance Act. Importantly, the 2018 Farm Bill preserved FDA authority to regulate products with cannabis or cannabis-derived compounds under the Federal Food, Drug, and Cosmetic (FD&C) Act and Section 351 of the Public Health Service Act.

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New Drug Approvals

SEPTEMBER 10, 2024

2] Vorasidenib was approved for medical use in the United States in August 2024. [2] 2] [3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation. [2]

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The Pharma Data

MAY 31, 2022

Without treatment, infants do not achieve these milestones in the natural history of the disease. “The priority review and subsequent approval of Evrysdi for babies under two months of age speaks to the urgent ongoing need for additional treatment options for babies with SMA,” said Levi Garraway, M.D.,

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Drug Target Review

AUGUST 29, 2024

MR : Chimeric antigen receptor T-cell (CAR-T) therapy is very effective in treating patients with B-cell lymphoma, leukemia, and multiple myeloma, where we have six FDA-approved drugs. What potential advantages does the CD5 modulation strategy offer over traditional CAR-T therapies?

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The Pharma Data

JANUARY 6, 2021

UNION therapeutics A/S is a privately held, clinical stage, pharmaceutical company dedicated to the development of novel treatments for inflammatory and infectious diseases. 2018)2: Menter MA, Armstrong AW, Gordon KB, Wu JJ. 2018 Feb;37(2S):S48-S51. About UNION therapeutics A/S. Sources 1: Li et al. Semin Cutan Med Surg.

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New Drug Approvals

SEPTEMBER 8, 2024

1] Palopegteriparatide was approved for medical use in the European Union in November 2023, [2] and in the United States in August 2024. [1] 5] The FDA granted the application for palopegteriparatide orphan drug and priority review designations. [5] 4] [6] The applicant for this medicinal product is Ascendis Pharma Bone Diseases A/S.

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Eye on FDA

DECEMBER 6, 2023

For those working closely with the development of new medicines for FDA approval, it can be informative respecting the future to look back at recent activity and take note of any potential changes from years past. There are two things of note from this year.

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Drug Target Review

JULY 5, 2024

Since brain organoids can be derived from either diseased or healthy patients, they can recapitulate human (patho)physiology making them a better choice compared to mammalian cell systems that often fail to recapitulate the human phenotype. 15(9):700-706 (2018) [link] Deshmukh V, et al. 12(3) (2023). link] Madhavan M, et al.

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The Pharma Data

JUNE 17, 2022

The application includes all indications covered by the reference medicine*, including rheumatoid arthritis, Crohn’s disease, ulcerative colitis, plaque psoriasis, and uveitis 1. Sandoz’ Hyrimoz 50 mg/mL was first approved by the European Commission in July 2018 and launched in several European countries shortly thereafter.

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The Pharma Data

DECEMBER 30, 2020

In December, Gilead shifted from bolstering its oncology business and strengthened its liver disease pipeline with the $1.4 AstraZeneca Dives into Rare Diseases: AstraZeneca has been in the spotlight for the COVID-19 vaccine candidate it developed with Oxford University. Andexxa, was approved by the U.S. But the U.K.-based

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Drug Discovery World

APRIL 14, 2023

He is being recognised for his revolutionary contributions to developing the first gene-edited cell-based therapy for cancer that involves the genetic re-engineering of a patient’s own T cells to combat their disease, and for demonstrating that adoptive T-cell therapy can induce remission and in some cases cure patients with advanced cancer.

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The Pharma Data

AUGUST 19, 2021

1 Disease response was evaluated using overall response rate (ORR), per Response Evaluation Criteria in Solid Tumors Version 1.1* (RECIST v1.1) RYBREVANT TM (amivantamab-vmjw) received accelerated approval by the U.S. In 2018, Janssen Biotech, Inc. Interstitial Lung Disease/Pneumonitis 7. as the primary endpoint.

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The Pharma Data

SEPTEMBER 28, 2021

Our goal is to still lead the way in developing transformative medicines for neuromuscular diseases. We are grateful for the partnerships that are helping us to develop new therapies for people impacted by these devastating rare diseases.”. About SMA SMA may be a severe, progressive neuromuscular disease which will be fatal.

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PLOS: DNA Science

JUNE 1, 2023

The newest FDA-approved gene therapy treats the severe, skin-peeling condition dystrophic epidermolysis bullosa (DEB). The gene treatment has been a long time coming, but it differs from the handful of other approved gene therapies: it isn’t a one-and-done. This DNA Science post from 2018 traces the history of the efforts.

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The Pharma Data

JANUARY 31, 2021

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. . CHENGDU, China , Feb. About Trimer-Tag© Technology.

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Metabolite Tales Blog

JANUARY 23, 2024

Metabolism of 2023 FDA Approved Small Molecules – PART 1 By Julia Shanu-Wilson 2023 was a fruitful year for drug approvals by the FDA, with a crop of 34 small molecules out of a total of 55 new drugs [1]. link] [19] FDA prescribing information for zilucoplan. link] [11] Zheng et al.,

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The Pharma Data

SEPTEMBER 9, 2020

Basel, 10 September 2020 – Roche (SIX: RO, ROG; OTCQX: RHHBY) will present new ENSPRYNG® (satralizumab) data on reducing relapse severity in the treatment of neuromyelitis optica spectrum disorder (NMOSD), a rare disease of the central nervous system. 0.86, p=0.015). A relapse was categorised as severe if it resulted in a change of ?

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Dark Matter Blog

AUGUST 27, 2020

In early August, risdiplam, an orally available small molecule that binds to a stem-loop structure in the SMN2 pre-mRNA, received FDA approval for the treatment of spinal muscular atrophy , a devastating genetic disease. In less severe disease, patients will reach adulthood and suffer from motor function deficiencies.

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The Pharma Data

DECEMBER 3, 2020

Many major disease targets are trimerization-dependent such as the tumor necrosis factor (TNF) superfamily (involved in extrinsic apoptosis, immune co-stimulation and inflammation) as well as enveloped RNA virus antigens responsible for entry into host cells. About Trimer-Tag © Technology. View source version on businesswire.com: [link].

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The Pharma Data

MARCH 30, 2021

– Migraine is a debilitating neurological disease affecting 39 million people in the U.S.[2] Migraine is a complex, chronic disease with attacks that are often incapacitating and can include headache pain as well as neurologic and autonomic symptoms. AbbVie anticipates a regulatory decision in late Q3 2021.

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The Pharma Data

JUNE 28, 2021

Migraine is a Disabling Neurological Disease that Affects More Than 8.4 Having this treatment approved in Japan will enable us to ultimately serve more patients and help them find the right treatment for this disabling, neurological disease.” 1,7 Although approximately 8.4 became a wholly owned affiliate of Amgen Inc.

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Drug Discovery World

JULY 8, 2024

While mRNA vaccines and RNA interference therapies have garnered much attention, a new class of RNA therapeutics known as aptamers is gaining momentum, particularly to address acute diseases. By silencing transthyretin (TTR) mRNA, patisiran decreases the production of TTR protein, the molecule responsible for causing the disease.

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Agency IQ

NOVEMBER 3, 2023

Synthetic biotics have been engineered to perform a variety of functions: “ Several classes of effectors have been engineered into bacteria, including enzyme pathways that metabolize disease-causing toxins, production of protein effectors, and production of small-molecule effectors,” according to a recent article on the topic.

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Drug Discovery World

AUGUST 10, 2023

In Nicotine dependence and smoking cessation, a paper available in the National Library of Medicine, co-authors shared that tobacco use is the single most preventable cause of death, disability and disease in the world and was projected to be the leading cause of death and disability across all developed and developing countries by 2020 1.

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The Pharma Data

JUNE 18, 2021

4,5 Between 20 and 46 percent of adults with atopic dermatitis have moderate to severe disease. 6 The range of symptoms pose significant physical, psychological and economic burden on individuals impacted by the disease. The approved dose for RINVOQ is 15 mg. Nat Rev Dis Primers 4, 1(2018). About RINVOQ ® (upadacitinib).

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The Pharma Data

JUNE 18, 2021

First approval of a conjugate vaccine that helps protect against 20 serotypes responsible for the majority of invasive pneumococcal disease and pneumonia, 1,2,3,4,5,6,7 including seven responsible for 40% of pneumococcal disease cases and deaths in the U.S. Following today’s FDA approval, the U.S. Jansen, Ph.D.,

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Agency IQ

JUNE 7, 2024

Dietary supplement products may only make “structure/function” claims – claims that the product affects the structure or function of the body – or claims of nutritional support, and may not make any claim that a supplement treats or prevents a specific disease. See AgencyIQ’s in-depth analysis of the FDA’s approach to CBD regulation here.

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The Pharma Data

JANUARY 24, 2021

Sairiyo is a biotechnology company focused on repurposing and developing improved formulations of naturally derived compounds for serious, rare, and life-threatening diseases with the aim to obtain European Medicines Evaluation Agency and U.S. Food and Drug Administration (“FDA”) approval.

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The Pharma Data

JUNE 18, 2021

BOTOX ® Cosmetic is the first and only product of its kind FDA-approved to treat three areas, temporarily improving the appearance of moderate to severe forehead lines, glabellar lines and crow’s feet in adults. Department of Health and Human Services FDA Approval Letter for BOTOX® Cosmetic for Glabellar Lines, 2002.

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Drug Target Review

JUNE 21, 2023

Most rare diseases are caused by a single gene defect, but severity can vary considerably among patients. Modifier genes can help explain that variability and can alter or even prevent disease onset and progression, making them appealing therapeutic targets. However, the identification of these genes is challenging.

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The Pharma Data

MAY 18, 2021

5 percent of the tumour area) as determined by an FDA-approved test or are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. Tecentriq’s indication was subsequently focused on PD-L1 high patients, who would benefit the most based on findings from the IMvigor130 study in 2018.

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The Pharma Data

FEBRUARY 8, 2021

Based on these data, ADT alone should no longer be considered sufficient for patients with advanced, castration-sensitive disease.”. Food and Drug Administration (FDA) approval for nmCRPC on February 14, 2018 and was approved for mCSPC on September 17, 2019. ERLEADA ® received U.S.

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The Pharma Data

JANUARY 14, 2021

Food and Drug Administration (FDA) approved Pfizer ’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. Manuel Esteban/Shutterstock. Most Read Today. Source link.

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The Pharma Data

AUGUST 31, 2021

As the treatment of genitourinary cancers becomes more complex, we continue to work with urologists and their teams to improve outcomes for patients across the continuum of disease,” said Craig Tendler, M.D., Monitor for signs and symptoms of ischemic heart disease and cerebrovascular disorders. 2] ERLEADA ® received U.S.

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Agency IQ

JULY 21, 2023

BY RACHEL COE, MSC JUL 18, 2023 11:14 PM CDT Oncology endpoints: The growing pains of an evolving field FDA approval of a drug or biologic relies on whether there is “substantial evidence” of safety and effectiveness derived from “adequate and well-controlled clinical investigations” according to the Federal Food, Drug, and Cosmetic (FD&C) Act.

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The Pharma Data

DECEMBER 27, 2020

This year has been difficult, but it has reinforced the importance of our mission: to treat and potentially even reverse the effects of serious diseases and conditions by advancing our novel cell therapy product candidates through clinical trials and into the hands of physicians.

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Agency IQ

MAY 17, 2024

The Creating Hope Reauthorization Act would reauthorize the FDA’s Rare Pediatric Disease Priority Review Voucher program. The markup hearing comes three months after the E&C Committee last held a hearing on 18 bills related to rare diseases, including all four of the above-mentioned bills. DORIS MATSUI (D-Calif.)

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