Last update 21 Mar 2024

NPRB

Atrial natriuretic peptide receptor type B

Last update 21 Mar 2024

Basic Info

Synonyms

acromesomelic dysplasia, Maroteaux type, AMDM, ANP-B

+ [16]

Introduction

Receptor for the C-type natriuretic peptide NPPC/CNP hormone. Has guanylate cyclase activity upon binding of its ligand. May play a role in the regulation of skeletal growth.

Drugs associated with NPRB

Vosoritide

Target

NPRB

Mechanism

NPRB agonists

Active Org.

BioMarin Pharmaceutical, Inc. [+3]

Originator Org.

BioMarin International Ltd.

Active Indication

Achondroplasia [+1]

Inactive Indication-

Drug Highest PhaseApproved

First Approval Ctry. / Loc.

EU [+3]

First Approval Date26 Aug 2021

Cenderitide

Target

NPRA x NPRB

Mechanism

NPRA agonists [+1]

Active Org.-

Originator Org.

Capricor Therapeutics, Inc.

Active Indication-

Inactive Indication

Chronic heart failure [+2]

Drug Highest PhaseDiscontinued

First Approval Ctry. / Loc.-

First Approval Date20 Jan 1800

Clinical Trials associated with NPRB

NCT05849389 / RecruitingPhase 2IIT

Vosoritide for Treatment of Short Stature in Girls With Turner Syndrome

Turner syndrome (TS) is characterized by a missing whole or part of the second sex chromosome in a phenotypic female, resulting in short stature due to haploinsufficiency of the short-stature homeobox-containing (SHOX) gene. Growth hormone (GH) is an approved therapy for this condition, although not associated with GH deficiency, and benefits are modest. Vosoritide, a C-type natriuretic peptide (CNP) analog, targets chondrocytes within the growth plate leading to increased cell proliferation and hypertrophy. We hypothesize that patients with TS and short stature will respond to vosoritide treatment leading to increased growth velocity. This study will enroll pre-pubertal girls with TS who are either naïve to GH or have had a poor response to GH therapy. All subjects will be treated with vosoritide for 12 months and will be assessed for safety monitoring and improvement in height outcomes. Annualized growth velocity (AGV) on vosoritide will be compared to AGV in the 6-18 months prior to initiation of vosoritide based on historical data available in the medical record. Subjects with a positive response to therapy will be given the option to continue in the extension phase of the study during which they will continue to receive vosoritide until growth cessation.

Start Date01 Mar 2024

Sponsor / Collaborator

Children's National Research Institute

NCT05813314 / TerminatedPhase 1

A Phase 1, Randomized, Open-Label, Sponsor-Open, Two-Sequence, Two-Period Crossover Study to Investigate the Bioequivalence of a Single Subcutaneous Dose of BMN 111 Administered Using Either a Vial and Syringe or Injector Pen in Healthy Adult Participants

The purpose of this study is to establish the bioequivalence of a single SC dose of BMN 111 administered using a vial and syringe versus using an injector pen.

Start Date15 Mar 2023

Sponsor / Collaborator

BioMarin Pharmaceutical, Inc.

NCT04554940 / Active, not recruitingPhase 2

A Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression Surgery

Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery

Start Date10 Oct 2020

Sponsor / Collaborator

BioMarin Pharmaceutical, Inc.

100 Clinical Results associated with NPRB

100 Translational Medicine associated with NPRB

0 Patents (Medical) associated with NPRB

1,693

Literatures (Medical) associated with NPRB

19 Feb 2024·Cell and tissue research

Paracrine and endocrine pathways of natriuretic peptides assessed by ligand-receptor mapping in the Japanese eel brain.

Article

Author: Tomoki Izumi ; Ami Saito ; Takanori Ida ; Takao Mukuda ; Yukitoshi Katayama ; Marty Kwok-Shing Wong ; Takehiro Tsukada

The natriuretic peptide (NP) family consists of cardiac NPs (ANP, BNP, and VNP) and brain NPs (CNPs) in teleosts. In addition to CNP1-4, a paralogue of CNP4 (named CNP4b) was recently discovered in basal teleosts including Japanese eel. Mammals have lost most Cnps during the evolution, but teleost cnps were conserved and diversified, suggesting that CNPs are important hormones for maintaining brain functions in teleost. The present study evaluated the potency of each Japanese eel CNP to their NP receptors (NPR-A, NPR-B, NPR-C, and NPR-D) overexpressed in CHO cells. A comprehensive brain map of cnps- and nprs-expressing neurons in Japanese eel was constructed by integrating the localization results obtained by in situ hybridization. The result showed that CHO cells expressing NPR-A and NPR-B induced strong cGMP productions after stimulation by cardiac and brain NPs, respectively. Regarding brain distribution of cnps, cnp1 is engaged in the ventral telencephalic area and periventricular area including the parvocellular preoptic nucleus (Pp), anterior/posterior tuberal nuclei, and periventricular gray zone of the optic tectum. cnp3 is found in the habenular nucleus and prolactin cells in the pituitary. cnp4 is expressed in the ventral telencephalic area, while cnp4b is expressed in the motoneurons in the medullary area. Such CNP isoform-specific localizations suggest that function of each CNP has diverged in the eel brain. Furthermore, the Pp lacking the blood-brain barrier expressed both npra and nprb, suggesting that endocrine and paracrine NPs interplay for regulating the Pp functions in Japanese eels.

06 Feb 2024·World journal of clinical cases

Effect of early stepwise cardiopulmonary rehabilitation on function and quality of life in sepsis patients.

Article

Author: Ming-Hui Zheng ; Wen-Jun Liu ; Juan Yang

BACKGROUND:

Sepsis, as a non-limiting host infection disease, can be accompanied by serious complications such as organ failure, which seriously threatens patient quality of life.

AIM:

To investigate the effect of early stepwise cardiopulmonary rehabilitation on cardiopulmonary function and quality of life in patients evacuated from mechanical ventilation with sepsis.

METHODS:

A total of 80 patients with sepsis who were hospitalized in our hospital from January 2021 to January 2022 were selected and divided into the observation group (n = 40) and the control group (n = 40) according to the random number table method. The observation group was treated with early stepwise cardiopulmonary rehabilitation, and the control group was treated with a conventional treatment regimen. Cardiac function indexes (central venous pressure, cardiac troponin I, B-type brain natriuretic peptide), lung function indicators (diaphragmatic mobility, changes in central venous oxygen saturation, oxygenation index), and quality of life (Quality of Life Evaluation Scale) were compared between the two groups after treatment.

RESULTS:

After treatment, the central venous pressure, diaphragm mobility, central venous oxygen saturation, oxygenation index, and Quality of Life Evaluation Scale scores in the observation group were higher than those in the control group, and the differences were statistically significant (P < 0.05). The observation group was less than that of the control group for other parameters, and the differences were statistically significant (P < 0.05).

CONCLUSION:

Early stepwise cardiopulmonary rehabilitation can effectively enhance cardiac and pulmonary function and improve the quality of life in patients evacuated from mechanical ventilation with sepsis.

27 Jan 2024·Journal of medical case reports

Treatment strategy for heart failure complicated with complete left bundle branch block and atrial tachycardia: a case report.

Article

Author: Jian Wang ; Qing-Qing Zhang ; Jie Lin ; Wei Yuan ; Li-Chun Wang ; Yu-Cheng Wu

BACKGROUND:

For patients with heart failure combined with complete left bundle branch block, cardiac resynchronization therapy is an important therapeutic method. If these patients also have atrial tachycardia, how to choose a treatment strategy deserves discussion.

CASE PRESENTATION:

A Chinese woman in her early 70s was admitted due to recurrent episodes of chest distress and asthma for 1 year. Physical and laboratory examinations showed filling of the jugular vein, lung rales, left enlargement of the heart boundary, edema of the lower limbs and elevation of N-terminal pro b-type natriuretic peptide. An electrocardiogram showed atrial tachycardia and a left bundle branch block. An echocardiography revealed enlargement of the left ventricle and left ventricular systolic dysfunction. After obtaining informed consent, the treatment strategy decided upon by the team was to use biventricular cardiac resynchronization therapy treatment and to not intervene for the atrial tachycardia, with left bundle branch area pacing as a backup. Due to twisted and narrow coronary vein branches, traditional biventricular pacing failed, and then, left bundle branch area pacing was attempted successfully. A follow-up echocardiography at 1 year showed improved systolic function. The outcomes for this patient are favorable, but the choice of interventional strategy is worthy of discussion in this case.

CONCLUSION:

For patients with heart failure combined with left bundle branch block and atrial tachycardia, left bundle branch area pacing can replace traditional biventricular pacing for cardiac resynchronization therapy treatment, and the therapeutic effect is significant. However, multiple factors need to be considered when formulating strategies, including whether there is bundle branch block under sinus rhythm, the success and recurrence rate of atrial tachycardia ablation, the response of cardiac resynchronization therapy, the costs of different strategies, and instrument implantation issues.

News (Medical) associated with NPRB

23 Jun 2023

·www.pharmaceutical-technology.com

MSD extends peptide collaboration with IRBM

Aidan McNamee IRBM claims its partnership with MSD has benefitted from recent advances in phage and mRNA display platforms. Source: Shutterstock / testing. Contract research organisation IRBM has announced an extension to its collaboration with US-based Merck & Co. (MSD) to develop peptide therapeutics. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. 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British (+1284) Virgin Islands - US (+1340) Wallis & Futuna (+681) Yemen (North)(+969) Yemen (South)(+967) Zambia (+260) Zimbabwe (+263) Country UK USA Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bonaire, Sint Eustatius and Saba Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos Islands Colombia Comoros Congo Democratic Republic of the Congo Cook Islands Costa Rica Côte d"Ivoire Croatia Cuba Curaçao Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-Bissau Guyana Haiti Heard Island and McDonald Islands Holy See Honduras Hong Kong Hungary Iceland India Indonesia Iran Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati North Korea South Korea Kuwait Kyrgyzstan Lao Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia Moldova Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Réunion Romania Russian Federation Rwanda Saint Helena, Ascension and Tristan da Cunha Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan Tajikistan Tanzania Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates US Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Vietnam British Virgin Islands US Virgin Islands Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Kosovo By downloading this case study, you acknowledge that GlobalData may share your information with GlobalData and that your personal data will be used as described in their Privacy Policy Submit Visit our Privacy Policy for more information about our services, how GlobalData may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report. IRBM began as part of MSD , in 2000, before being spun off in 2010. Following the separation, the companies continued to work together on drug discovery, with the collaboration being expanded in 2020 to develop peptide therapeutics for coronavirus. Recommended Reports Reports Atrial Natriuretic Peptide Receptor 2 (Atrial Natriuretic Peptide Receptor Type B or Guanylate Cy... GlobalData Reports Glycylpeptide N Tetradecanoyltransferase (Myristoyl CoA:Protein N Myristoyltransferase or Peptide... GlobalData View all Advances in phage and mRNA display platforms have accelerated peptide development in recent years. In particular, IRBM claims it has made major progress with half-life extension strategies and utilising formulation enhancers to support oral route administrations. IRBM told Pharmaceutical Technology that AI is beginning to play a part in peptide discovery, and that radiotheranostics was a rapidly growing area of interest. Italy-based IRBM draws upon expertise in peptide drug development to bring new candidates to fruition. It utilises a platform that covers the entire range of activities required to discover and develop novel peptide hits into potential drug leads. One candidate discovered through the collaboration with MSD is MK-0616, a macrocyclic peptide designed to treat hypercholesterolemia by inhibiting the PCSK9 protein associated with high levels of cholesterol. While there are available treatments in this area, costly injectables and statins are poorly tolerated by many patients. In March, MSD presented positive results from a Phase II trial of MK-0616, where the peptide drug reduced low-density lipoprotein (LDL) cholesterol by more than 60% at the highest dose. With a Phase III trial planned for H2 2023, MSD and IRBM’s collaboration could fill an important niche in providing easier, cost-effective treatment to patients. In a 22 June press release , IRBM peptide chemistry senior director Elisabetta Bianchi celebrated the extension, claiming that the collaboration had already produced “outstanding achievements” in delivering orally available peptide candidates. IRBM has also aided in the discovery of MK-1462, a potential candidate for obesity and diabetes. Whilst this is in early development, obesity treatments have proven promising for other pharma heavyweights. Novo Nordisk ’s Wegovy (semaglutide) is already a success in obesity treatments, and Eli Lilly ’s Mounjaro (tirzepatide) is likely to be a strong competitor . According to GlobalData analysis, the market for obesity drugs across the seven major markets (US, France, Germany, Italy, Spain, UK, and Japan) is projected to hit $37bn by 2031, up from $2.4bn in 2021. GlobalData is the parent company of Pharmaceutical Technology. Free Report How is the Biopharmaceutical industry evolving? 2021 was a year of continued innovation and change in the Biopharmaceutical industry. As the COVID-19 pandemic continues to take its toll on businesses worldwide, it’s time to look for new ways to create value, prepare for the future, and remain competitive in the ever-changing landscape. GlobalData’s expansive report examines the business environment and trends that shape the Biopharmaceutical industry. We highlight the most impactful emerging technologies, as well as the industry, regulatory, and macroeconomic factors that influence growth prospects. Access the report to: Benchmark the impact of major themes on the Biopharmaceutical industry. Gain a deeper "on the ground" perspective through exclusive opinions and analysis from industry respondents. Evaluate the effects of COVID-19 on the sector. Download the full report to understand what to expect and how to align your strategies for success. 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Bissau (+245) Guyana (+592) Haiti (+509) Honduras (+504) Hong Kong (+852) Hungary (+36) Iceland (+354) India (+91) Indonesia (+62) Iran (+98) Iraq (+964) Ireland (+353) Israel (+972) Italy (+39) Jamaica (+1876) Japan (+81) Jordan (+962) Kazakhstan (+7) Kenya (+254) Kiribati (+686) Korea North (+850) Korea South (+82) Kuwait (+965) Kyrgyzstan (+996) Laos (+856) Latvia (+371) Lebanon (+961) Lesotho (+266) Liberia (+231) Libya (+218) Liechtenstein (+417) Lithuania (+370) Luxembourg (+352) Macao (+853) Macedonia (+389) Madagascar (+261) Malawi (+265) Malaysia (+60) Maldives (+960) Mali (+223) Malta (+356) Marshall Islands (+692) Martinique (+596) Mauritania (+222) Mayotte (+269) Mexico (+52) Micronesia (+691) Moldova (+373) Monaco (+377) Mongolia (+976) Montserrat (+1664) Morocco (+212) Mozambique (+258) Myanmar (+95) Namibia (+264) Nauru (+674) Nepal (+977) Netherlands (+31) New Caledonia (+687) New Zealand (+64) Nicaragua (+505) Niger (+227) Nigeria (+234) Niue (+683) Norfolk Islands (+672) Northern Marianas (+670) Norway (+47) Oman (+968) Palau (+680) Panama (+507) Papua New Guinea (+675) Paraguay (+595) Peru (+51) Philippines (+63) Poland (+48) Portugal (+351) Puerto Rico (+1787) Qatar (+974) Reunion (+262) Romania (+40) Russia (+7) Rwanda (+250) San Marino (+378) Sao Tome & Principe (+239) Saudi Arabia (+966) Senegal (+221) Serbia (+381) Seychelles (+248) Sierra Leone (+232) Singapore (+65) Slovak Republic (+421) Slovenia (+386) Solomon Islands (+677) Somalia (+252) South Africa (+27) Spain (+34) Sri Lanka (+94) St. Helena (+290) St. Kitts (+1869) St. Lucia (+1758) Sudan (+249) Suriname (+597) Swaziland (+268) Sweden (+46) Switzerland (+41) Syria (+963) Taiwan (+886) Tajikstan (+7) Thailand (+66) Togo (+228) Tonga (+676) Trinidad & Tobago (+1868) Tunisia (+216) Turkey (+90) Turkmenistan (+7) Turkmenistan (+993) Turks & Caicos Islands (+1649) Tuvalu (+688) Uganda (+256) Ukraine (+380) United Arab Emirates (+971) Uruguay (+598) Uzbekistan (+7) Vanuatu (+678) Vatican City (+379) Venezuela (+58) Vietnam (+84) Virgin Islands - British (+1284) Virgin Islands - US (+1340) Wallis & Futuna (+681) Yemen (North)(+969) Yemen (South)(+967) Zambia (+260) Zimbabwe (+263) Country UK USA Afghanistan Åland Islands Albania Algeria American Samoa Andorra Angola Anguilla Antarctica Antigua and Barbuda Argentina Armenia Aruba Australia Austria Azerbaijan Bahamas Bahrain Bangladesh Barbados Belarus Belgium Belize Benin Bermuda Bhutan Bolivia Bonaire, Sint Eustatius and Saba Bosnia and Herzegovina Botswana Bouvet Island Brazil British Indian Ocean Territory Brunei Darussalam Bulgaria Burkina Faso Burundi Cambodia Cameroon Canada Cape Verde Cayman Islands Central African Republic Chad Chile China Christmas Island Cocos Islands Colombia Comoros Congo Democratic Republic of the Congo Cook Islands Costa Rica Côte d"Ivoire Croatia Cuba Curaçao Cyprus Czech Republic Denmark Djibouti Dominica Dominican Republic Ecuador Egypt El Salvador Equatorial Guinea Eritrea Estonia Ethiopia Falkland Islands Faroe Islands Fiji Finland France French Guiana French Polynesia French Southern Territories Gabon Gambia Georgia Germany Ghana Gibraltar Greece Greenland Grenada Guadeloupe Guam Guatemala Guernsey Guinea Guinea-Bissau Guyana Haiti Heard Island and McDonald Islands Holy See Honduras Hong Kong Hungary Iceland India Indonesia Iran Iraq Ireland Isle of Man Israel Italy Jamaica Japan Jersey Jordan Kazakhstan Kenya Kiribati North Korea South Korea Kuwait Kyrgyzstan Lao Latvia Lebanon Lesotho Liberia Libyan Arab Jamahiriya Liechtenstein Lithuania Luxembourg Macao Macedonia, The Former Yugoslav Republic of Madagascar Malawi Malaysia Maldives Mali Malta Marshall Islands Martinique Mauritania Mauritius Mayotte Mexico Micronesia Moldova Monaco Mongolia Montenegro Montserrat Morocco Mozambique Myanmar Namibia Nauru Nepal Netherlands New Caledonia New Zealand Nicaragua Niger Nigeria Niue Norfolk Island Northern Mariana Islands Norway Oman Pakistan Palau Palestinian Territory Panama Papua New Guinea Paraguay Peru Philippines Pitcairn Poland Portugal Puerto Rico Qatar Réunion Romania Russian Federation Rwanda Saint Helena, Ascension and Tristan da Cunha Saint Kitts and Nevis Saint Lucia Saint Pierre and Miquelon Saint Vincent and The Grenadines Samoa San Marino Sao Tome and Principe Saudi Arabia Senegal Serbia Seychelles Sierra Leone Singapore Slovakia Slovenia Solomon Islands Somalia South Africa South Georgia and The South Sandwich Islands Spain Sri Lanka Sudan Suriname Svalbard and Jan Mayen Swaziland Sweden Switzerland Syrian Arab Republic Taiwan Tajikistan Tanzania Thailand Timor-Leste Togo Tokelau Tonga Trinidad and Tobago Tunisia Turkey Turkmenistan Turks and Caicos Islands Tuvalu Uganda Ukraine United Arab Emirates US Minor Outlying Islands Uruguay Uzbekistan Vanuatu Venezuela Vietnam British Virgin Islands US Virgin Islands Wallis and Futuna Western Sahara Yemen Zambia Zimbabwe Kosovo By downloading this case study, you acknowledge that GlobalData may share your information with GlobalData and that your personal data will be used as described in their Privacy Policy Submit Visit our Privacy Policy for more information about our services, how GlobalData may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. Thank you. Please check your email to download the Report.

Clinical ResultAcquisitionPhase 2Phase 3

25 Apr 2023

·www.pharmaceutical-technology.com

3BP and Novartis strike licensing agreement for FAP-targeting peptide technology

3B Pharmaceuticals (3BP) is a German biotechnology company developing radiopharmaceutical drugs and diagnostics for oncology indications. Credit: stevepb from Pixabay. German biotechnology firm 3B Pharmaceuticals (3BP) has entered into a licensing agreement with Novartis Innovative Therapies for its fibroblast activation protein (FAP)-targeting peptide technology. The deal provides Novartis with exclusive rights across the globe to create and commercialise therapeutic and imaging solutions for 3BP’s entire FAP-targeting peptide technology, including FAP-2286. Recommended Reports Reports Atrial Natriuretic Peptide Receptor 2 (Atrial Natriuretic Peptide Receptor Type B or Guanylate Cy... GlobalData Reports Clostridium difficile Toxin B (toxB or EC 3.4.22.) Drugs in Development by Therapy Areas and Indi... GlobalData View all 3BP receives an initial payment of $40m, and $425m as development, regulatory and commercial milestone payments. Tiered royalties on net sales are also included in the deal. 3BP continues to hold specific rights to develop its FAP-targeting imaging technology for diagnostic applications. FAP-2286 is being investigated as a peptide-targeted radionuclide therapy. It is capable of targeting FAP, a significant theranostic target with expression across various types of cancers. FAP-2286 marks the first peptide-targeted radioligand therapy aiming at FAP to enter clinical development, along with the respective imaging agent. The therapy candidate is currently under evaluation in the Phase l LuMIERE clinical trial. 3BP nuclear medicine and imaging head Dr Christiane Smerling stated: “We believe the new agreement with Novartis is an ideal partnership for the further clinical development of FAP-2286 for the benefit of patients with many different types of cancer.” In 2019, 3BP reached a licence and collaboration deal with Clovis Oncology to support the development of a peptide-targeted radiotherapy and imaging agent targeting FAP.

License out/inPhase 1PDC

19 Nov 2021

·endpts.com

FDA opens up an ethical can of worms with accelerated approval for first drug for underlying genetic cause of dwarfism

The FDA on Friday signed off on an accelerated approval for BioMarin’s Voxzogo (vosoritide) injection, the first treatment to target the underlying genetics of dwarfism, which can increase the height of children five years of age and older with the condition. The injection, which Biomarin said will have an annual net price of $240,000, works by binding to a specific receptor called the natriuretic peptide receptor-B, which reduces the growth regulation gene’s activity and stimulates bone growth in children. “Today’s approval fulfills an unmet medical need for more than 10,000 children in the United States,” said Theresa Kehoe, director of FDA’s Division of General Endocrinology. The approval is based on a Phase III placebo-controlled trial in which 121 participants were randomly assigned to receive either Voxzogo injections or a placebo. “Researchers measured the participants’ annualized growth velocity, or rate of height growth, at the end of the year. Participants who received Voxzogo grew an average 1.57 centimeters taller compared to those who received a placebo,” the FDA said. A condition of the accelerated approval is that BioMarin must run a post-marketing study that will assess the final adult height. Jean-Jacques Bienaimé, chairman and CEO of BioMarin, said in a statement, “Voxzogo is a medical first that is rooted in BioMarin’s focus on molecular genetics and targets the underlying cause of the condition. More than a decade of scientific research underpins the medical advance that Voxzogo represents. We thank the FDA for recognizing its value as the first therapeutic treatment option for children with achondroplasia.” But there’s an ethical question at the heart of the approval too. According to STAT News , organizations such as Little People of America have long sought to advocate for fairness while noting that achondroplasia, the most common form of dwarfism, doesn’t preclude them from a fulfilling life, and drugs like Voxzogo could be a threat to the community they’ve built.

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NPRB

Atrial natriuretic peptide receptor type B

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