Orphan drugs

Excel Diagnostics and Nuclear Oncology Center Announces FDA Authorization of IND for 225Ac-PSMA I&T Targeted Alpha Therapy for Castration Resistant Prostate Cancer

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水曜日, 8月 4, 2021

Branches of biology, Health sciences, Chemistry, Radiopharmaceuticals, Orphan drugs, Chelating agents, Macrocycles, Glutamate carboxypeptidase II, Proteases, MCRPC, DOTA-TATE, Prostate cancer

Houston, TX, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Excel Diagnostics and Nuclear Oncology Center (EDNOC), a premier Diagnostic Imaging and Radioligand Therapy center in Houston, Texas announces approval of its physicians sponsored Investigational New Drug (IND) application for providing Targeted Alpha Therapy (TAT) with 225Ac-PSMA I&T for metastatic Castration-Resistant Prostate Cancer (mCRPC).

Key Points:

Houston, TX, Aug. 04, 2021 (GLOBE NEWSWIRE) -- Excel Diagnostics and Nuclear Oncology Center (EDNOC), a premier Diagnostic Imaging and Radioligand Therapy center in Houston, Texas announces approval of its physicians sponsored Investigational New Drug (IND) application for providing Targeted Alpha Therapy (TAT) with 225Ac-PSMA I&T for metastatic Castration-Resistant Prostate Cancer (mCRPC).
Excel Diagnostics and Nuclear Oncology Center has been a pioneer in bringing new targeted Radioligand Therapies such as Lu-177 DOTATATE, and Lu-177 PSMA to serve our patients in the United States.
Highly encouraging results have been reported by using Targeted Alpha Therapy (TAT) Radiopharmaceuticals in conditions such as neuroendocrine, prostate, or hematological malignancies.
In April of 2020, Excel passed FDA inspection of one of its investigational drugs with no deficiencies or citations.

F-star Therapeutics Announces Collaboration with MSD to Evaluate FS120 in Combination with KEYTRUDA

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水曜日, 8月 4, 2021

Clinical medicine, Cancer treatments, Medicine, Orphan drugs, Antineoplastic drugs, Breakthrough therapy, Cancer immunotherapy, Immune system, Pembrolizumab, Programmed cell death protein 1, Antibody, Preclinical development

Under the terms of the agreement, MSD will supply KEYTRUDA for a combination arm that will be included in theadaptive Phase 1 clinical protocol of FS120, sponsored by F-star,thatwas initiatedin December of 2020.

Key Points:

Under the terms of the agreement, MSD will supply KEYTRUDA for a combination arm that will be included in theadaptive Phase 1 clinical protocol of FS120, sponsored by F-star,thatwas initiatedin December of 2020.
FS120 is currently being exploredas a monotherapy in dose escalation (NCT04648202) , including the evaluation of pharmacokinetics and pharmacodynamics in patients with advanced cancer.
FS120 will also be evaluated in combination withKEYTRUDA,with the potential for early demonstration of clinical activity in specifictumorsubtypes.
In preclinical studies, FS120 has demonstrated strong additive effects in combination with PD-1 monoclonal antibodies.

UroGen Pharma Reports Second Quarter 2021 Financial Results and Recent Corporate Developments

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水曜日, 8月 4, 2021

The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC.

Key Points:

The U.S. FDA previously granted Orphan Drug, Fast Track, and Breakthrough Therapy Designations to Jelmyto for the treatment of LG-UTUC.
You should not receive JELMYTO if you have a hole or tear (perforation) of your bladder or upper urinary tract.
Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with JELMYTO.
JELMYTO and UroGen are registered trademarks of UroGen Pharma, Ltd.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210804005328/en/

Insilico Medicine Announces the Preclinical Candidate for Kidney Fibrosis Discovered Using End-to-End Artificial Intelligence Engine

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水曜日, 8月 4, 2021

Kidney diseases, Chronic kidney disease, Organ failure, Orphan drugs

The preclinical candidate has the desirable pharmacological properties, pharmacokinetic profile, and demonstrated highly promising results in in-vitro and in-vivo preclinical studies.

Key Points:

The preclinical candidate has the desirable pharmacological properties, pharmacokinetic profile, and demonstrated highly promising results in in-vitro and in-vivo preclinical studies.
"Kidney fibrosis is the common pathogenesis in the progression of chronic kidney disease ("CKD") and is a major unmet medical need.
Approximately 850 million people worldwide , almost a billion, have kidney disease often being driven by or associated with kidney fibrosis.
Repeating that success, Insilico Medicine leveraged its PandaOmics to develop the target hypothesis for kidney fibrosis, and used Chemistry42 to generate compounds with drug-like properties.

Insilico Medicine Announces the Preclinical Candidate for Kidney Fibrosis Discovered Using End-to-End Artificial Intelligence Engine

Retrieved on:

水曜日, 8月 4, 2021

Kidney diseases, Chronic kidney disease, Organ failure, Orphan drugs

The preclinical candidate has the desirable pharmacological properties, pharmacokinetic profile, and demonstrated highly promising results in in-vitro and in-vivo preclinical studies.

Key Points:

The preclinical candidate has the desirable pharmacological properties, pharmacokinetic profile, and demonstrated highly promising results in in-vitro and in-vivo preclinical studies.
"Kidney fibrosis is the common pathogenesis in the progression of chronic kidney disease ("CKD") and is a major unmet medical need.
Approximately 850 million people worldwide , almost a billion, have kidney disease often being driven by or associated with kidney fibrosis.
Repeating that success, Insilico Medicine leveraged its PandaOmics to develop the target hypothesis for kidney fibrosis, and used Chemistry42 to generate compounds with drug-like properties.

NaviFUS begins FUS combined with radiotherapy clinical trial for the treatment of terminal primary brain tumors

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水曜日, 8月 4, 2021

Drugs, Health, Clinical medicine, Orphan drugs, Medical physics, Bevacizumab, Brain tumor, Radiation therapy, Breast cancer

TAIPEI, Aug. 4, 2021 /PRNewswire/ -- NaviFUS' focused ultrasound (FUS) therapy system (NaviFUS®) was recently approved for use in a new clinical trial by the Taiwan Food and Drug Administration (TFDA). It will be initiated shortly at Linkou Chang Gung Memorial Hospital. The trial will investigate the "synergy" that FUS-mediated opening of the blood-brain barrier (BBB) has on improving the therapeutic effect of radiotherapy treatment in patients with end-stage primary brain tumors. Despite suffering from repeating surgeries, radiotherapy, and chemotherapy, most of these patients will eventually face tumor recurrence. Currently, while there may not be any effective treatments guaranteed to prolong survival, NaviFUS hopes that this upcoming clinical trial can result in a new, low-risk, and "Patient Friendly" option for patients who have already exhausted first and second-line treatments or failed radiotherapy treatment previously. If this combined treatment can safely enhance the effect of radiotherapy, NaviFUS' believes this treatment can also be extended to metastatic brain tumors from lung cancer, breast cancer, etc. The market potential for this treatment is expected to be more than $2 billion US dollars.

Key Points:

TAIPEI, Aug. 4, 2021 /PRNewswire/ -- NaviFUS' focused ultrasound (FUS) therapy system (NaviFUS) was recently approved for use in a new clinical trial by the Taiwan Food and Drug Administration (TFDA).
The trial will investigate the "synergy" that FUS-mediated opening of the blood-brain barrier (BBB) has on improving the therapeutic effect of radiotherapy treatment in patients with end-stage primary brain tumors.
If this combined treatment can safely enhance the effect of radiotherapy, NaviFUS' believes this treatment can also be extended to metastatic brain tumors from lung cancer, breast cancer, etc.
At the same time, NaviFUS is conducting a FUS-mediated BBB opening combined with bevacizumab (Avastin) clinical trial at Linkou Chang Gung Memorial Hospital.

NaviFUS begins FUS combined with radiotherapy clinical trial for the treatment of terminal primary brain tumors

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水曜日, 8月 4, 2021

Clinical medicine, Medicine, Health, Orphan drugs, Medical physics, Brain tumor, Oncology, Bevacizumab, Radiation therapy, Breast cancer, Metástasis, Cancer

The trial will investigate the "synergy" that FUS-mediated opening of the blood-brain barrier (BBB) has on improving the therapeutic effect of radiotherapy treatment in patients with end-stage primary brain tumors.

Key Points:

The trial will investigate the "synergy" that FUS-mediated opening of the blood-brain barrier (BBB) has on improving the therapeutic effect of radiotherapy treatment in patients with end-stage primary brain tumors.
If this combined treatment can safely enhance the effect of radiotherapy, NaviFUS' believes this treatment can also be extended to metastatic brain tumors from lung cancer, breast cancer, etc.
At the same time, NaviFUS is conducting a FUS-mediated BBB opening combined with bevacizumab (Avastin) clinical trial at Linkou Chang Gung Memorial Hospital.
"If these two non-invasive FUS treatments for brain tumors can be successfully developed, they can offer non-invasive treatment options with improved efficacy and minimal side effects."

Optime Care Offers Patient-First Humatin Total Care Program, Maximizes Therapeutic Benefits of Humatin™ for Hepatic Encephalopathy

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火曜日, 8月 3, 2021

Practice Management, Pharmaceutical, Managed care, Health, Data management, Infectious diseases, Hospitals, Technology, Brain disorders, Medical specialties, Anatomy, Branches of biology, Hepatology, Coma, Hepatic encephalopathy, Encephalopathy, Orphan drugs, Paromomycin, Liver

Optime Care , a nationally recognized pharmacy, distribution and patient management organization maximizing therapeutic opportunities for the treatment of orphan and rare disorders, announces the availability of Humatin (paromomycin sulfate) exclusively through Humatin Total Care .

Key Points:

Optime Care , a nationally recognized pharmacy, distribution and patient management organization maximizing therapeutic opportunities for the treatment of orphan and rare disorders, announces the availability of Humatin (paromomycin sulfate) exclusively through Humatin Total Care .
Humatin Total Care enables physicians to maximize the therapeutic opportunity for treating hepatic encephalopathy.
In the most severe form of hepatic encephalopathy, affected individuals may develop marked confusion or disorientation, amnesia, stupor or coma.
Quill concludes, As part of our patient-first focus, Humatin Total Care helps every stakeholder group impacted by this disease.

Marinus Pharmaceuticals Submits New Drug Application (NDA) to FDA for Ganaxolone for the Treatment of Seizures Associated with CDKL5 Deficiency Disorder and Provides Pipeline Update

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火曜日, 8月 3, 2021

Biotechnology, FDA, Pharmaceutical, Health, Chemical compounds, Anticonvulsants, GABAA receptor positive allosteric modulators, Orphan drugs, Ganaxolone, Pregnanes, Sedatives, Health care, Health, CDKL5 Deficiency Disorder, Ganaxolone, Marinus Pharmaceuticals, CDKL5 DEFICIENCY DISORDER, GANAXOLONE, MARINUS PHARMACEUTICALS

We look forward to working with the FDA in their review process and remain focused on preparing for the anticipated U.S. launch.

Key Points:

We look forward to working with the FDA in their review process and remain focused on preparing for the anticipated U.S. launch.
Ganaxolone was generally well-tolerated with a safety profile consistent with previous clinical trials, with the most frequent adverse event being somnolence.
Marinus received Rare Pediatric Disease (RPD) Designation from the FDA for ganaxolone for the treatment of seizures associated with CDD in July 2020.
Further details are available in Marinus press release distributed August 3, 2021 (Marinus Pharmaceuticals Announces Exclusive Collaboration with Orion Corporation for European Commercialization of Ganaxolone).

Nordic Nanovector Provides Update on PARADIGME, its Phase 2b Pivotal Trial with Betalutin® in R/R Follicular Lymphoma

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水曜日, 8月 4, 2021

Drugs, Orphan drugs, Health care, Health sciences, Rituximab, CD20, Breakthrough therapy

OSLO, Norway, Aug. 4, 2021 /PRNewswire/ -- Nordic Nanovector ASA (OSE: NANOV) provides an update on the timeline for PARADIGME, its ongoing pivotal Phase 2b trial of Betalutin (177Lu lilotomab satetraxetan) in 3rd-line relapsed rituximab/anti-CD20 refractory follicular lymphoma (3L R/R FL).

Key Points:

OSLO, Norway, Aug. 4, 2021 /PRNewswire/ -- Nordic Nanovector ASA (OSE: NANOV) provides an update on the timeline for PARADIGME, its ongoing pivotal Phase 2b trial of Betalutin (177Lu lilotomab satetraxetan) in 3rd-line relapsed rituximab/anti-CD20 refractory follicular lymphoma (3L R/R FL).
The Company will host a live webcast and Q&A on Thursday, 5 August 2021, at 08.30 CEST.
A link to the webcast will beavailable in the afternoon on Wednesday, 4 August on www.nordicnanovector.com .
In addition, the Company confirms it will invest no further funds in its Archer-1 Phase 1b trial investigating Betalutin in combination with rituximab in 2nd-line FL.