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Longer titles found: Orphan Drug Act of 1983 (view)

searching for Orphan drug 103 found (589 total)

Wild-type transthyretin amyloid (1,240 words) [view diff] exact match in snippet view article find links to article

the drug may have activity, the U.S. FDA in 2013 granted tolcapone "orphan drug status" in studies aiming at the treatment of transthyretin familial

Branaplam (374 words) [view diff] exact match in snippet view article find links to article

disease. In 2021, U.S. Food and Drug Administration (FDA) granted an orphan drug status to branaplam for treatment of Huntington’s disease, and Novartis

Arimoclomol (841 words) [view diff] exact match in snippet view article find links to article

Medicines Agency (EMA) and U.S. Food & Drug Administration (FDA) granted orphan drug designation to arimoclomol as a potential treatment for Niemann-Pick

TOL101 (766 words) [view diff] exact match in snippet view article find links to article

clinical trial in renal transplant patients.[when?][1] TOL101 was granted "orphan drug" status [2] by the U.S. Food and Drug Administration for the treatment

Niridazole (268 words) [view diff] exact match in snippet view article find links to article

Mittal N, Anupurba S (November 2007). "Ethylcellulose inserts of an orphan drug for periodontitis: preparation, in vitro, and clinical studies". Drug

Catalyst Pharmaceuticals (1,467 words) [view diff] exact match in snippet view article find links to article

development of amifampridine and its phosphate has brought attention to orphan drug policies that grant market exclusivity as an incentive for companies

Zoptarelin doxorubicin (762 words) [view diff] exact match in snippet view article find links to article

May 2017. The U.S. Food and Drug Administration (FDA) has granted it orphan drug status for ovarian cancer and endometrial cancer. Promising results have

Iclaprim (1,069 words) [view diff] exact match in snippet view article find links to article

disease product status for iclaprim. In September 2017, the FDA granted orphan drug status to iclaprim for the treatment of Staphylococcus aureus lung infections

Ezutromid (1,046 words) [view diff] exact match in snippet view article find links to article

Agency has given ezutromid an orphan drug designation. The FDA Office of Orphan Products and Development offers an Orphan Drug Designation program (ODD) that

Dexrazoxane (881 words) [view diff] exact match in snippet view article find links to article

anthracyclines are anticipated. Dexrazoxane was designated by the US FDA as an orphan drug for "prevention of cardiomyopathy for children and adults 0 through 16

Niemann–Pick disease, type C (5,123 words) [view diff] exact match in snippet view article find links to article

feeding tube). In 2014, the European Medicines Agency (EMA) granted orphan drug designation to arimoclomol for the treatment of Niemann–Pick type C.

ALK inhibitor (1,951 words) [view diff] exact match in snippet view article find links to article

RXDX-101, in the U.S. orphan drug designation and rare pediatric disease designation for the treatment of neuroblastoma and orphan drug designation for treatment

Miltefosine (3,583 words) [view diff] exact match in snippet view article find links to article

water entering the nose during a plunge in contaminated water. It has orphan drug status in the United States for acanthamoeba keratitis and primary amebic

Timothy Coté (825 words) [view diff] case mismatch in snippet view article find links to article

his time leading the OOPD, Coté was "instrumental in implementing the Orphan Drug Act" oversaw the approval of approximately 680 orphan designations. Coté

Cutaneous T-cell lymphoma (1,104 words) [view diff] exact match in snippet view article find links to article

phosphorylase) In 2010, the U.S. Food and Drug Administration granted orphan drug designation for naloxone lotion as a treatment for pruritus in cutaneous

Ataxia–telangiectasia (10,516 words) [view diff] case mismatch in snippet view article find links to article

Retrieved 2019-08-01. "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2019-08-01. "Search Orphan Drug Designations and Approvals"

GM2 gangliosidoses (1,215 words) [view diff] exact match in snippet view article find links to article

(Oxford, United Kingdom). N-Acetyl-Leucine has been granted multiple orphan drug designations from the U.S. Food & Drug Administration (FDA) and the European

OBI Pharma, Inc. (240 words) [view diff] case mismatch in snippet view article find links to article

Taiwan. In 2018 it announced that its drug, OBI-3424, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for

Congenital myasthenic syndrome (1,498 words) [view diff] exact match in snippet view article find links to article

4-diaminopyridine (amifampridine phosphate) is under development as an orphan drug for CMS and is available to eligible patients at no cost under an expanded

Interferon beta-1a (2,727 words) [view diff] case mismatch in snippet view article find links to article

company, originally under competition protection in the US under the Orphan Drug Act. Avonex is sold in three formulations, a lyophilized powder requiring

Interferon beta-1a (2,727 words) [view diff] case mismatch in snippet view article find links to article

company, originally under competition protection in the US under the Orphan Drug Act. Avonex is sold in three formulations, a lyophilized powder requiring

Marathon Pharmaceuticals (1,007 words) [view diff] case mismatch in snippet view article find links to article

diseases as outlined by the U.S. Food and Drug Administration’s (FDA) Orphan Drug Act. The Act defines rare diseases as impacting fewer than 200,000 patients

Tolcapone (2,140 words) [view diff] exact match in snippet view article find links to article

the drug may have activity, the U.S. FDA in 2013 granted tolcapone "orphan drug status" in studies aiming at the treatment of transthyretin familial

Amrubicin (288 words) [view diff] case mismatch in snippet view article find links to article

[dead link] Celgene Corporation (2008). "Amrubicin(R) Receives FDA Orphan Drug Designation for the Treatment of Small Cell Lung Cancer". Archived from

Neuromuscular junction (4,031 words) [view diff] exact match in snippet view article find links to article

4-Diaminopyridine, the first-line treatment for LEMS, is under development as an orphan drug for CMS in the US, and available to eligible patients under an expanded

Advanced Accelerator Applications (1,540 words) [view diff] exact match in snippet view article find links to article

diagnose somatostatin receptor-positive NET lesions. Each kit has received orphan drug designation from both the EMA and the FDA. 99MTc-rhAnnexin V-128, a SPECT

Familial hypercholesterolemia (5,456 words) [view diff] exact match in snippet view article find links to article

transfer protein, was approved by the US FDA in December 2012 as an orphan drug for the treatment of homozygous familial hypercholesterolemia. In January

Lipid-lowering agent (1,251 words) [view diff] exact match in snippet view article find links to article

Retrieved 31 January 2013 Staff (29 January 2013) FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder U.S. Food and Drug Administration

Niemann–Pick disease (3,076 words) [view diff] exact match in snippet view article find links to article

therapeutic strategy. In 2014, the European Medicines Agency granted orphan drug designation to arimoclomol for the treatment of Niemann–Pick type C.

ViroPharma (760 words) [view diff] case mismatch in snippet view article find links to article

Makiko (11 November 2013). "Shire Buys ViroPharma for $4.2 Billion to Add Orphan Drug". Bloomberg. Pollack, Andrew (9 November 2005). "Crucial Antibiotic Rescues

Glycerol phenylbutyrate (274 words) [view diff] exact match in snippet view article find links to article

on 29 June 2017. Retrieved 18 February 2018. Herder M (April 2016). "Orphan drug incentives in the pharmacogenomic context: policy responses in the USA

Arrowhead Pharmaceuticals (340 words) [view diff] case mismatch in snippet view article find links to article

deficiency In clinic, phase 3 Orphan Drug designation, partnered with Takeda ARO-APOC3 Hypertriglyceridemia Phase 2, 3 Orphan Drug designation, Fast Track Designation

Shire (pharmaceutical company) (3,016 words) [view diff] case mismatch in snippet view article

Retrieved 26 March 2016. "Shire Buys ViroPharma for $4.2 Billion to Add Orphan Drug". Bloomberg.com. Bloomberg. 11 November 2013. Archived from the original

Olinciguat (322 words) [view diff] exact match in snippet view article find links to article

stimulator that was in development for sickle cell anemia. After receiving orphan drug status in 2018 and completing a phase II trial, its development for sickle

Cannabis in South Korea (1,471 words) [view diff] case mismatch in snippet view article find links to article

Prior to obtaining the drug, patients are required to apply to the Korea Orphan Drug Center, a government body facilitating patient access to rare medicines

Envafolimab (86 words) [view diff] exact match in snippet view article find links to article

(dMMR) solid tumours. Envafolimab (KN035) has obtained the U.S. FDA's orphan drug designation for advanced biliary tract cancer. "Envafolimab - Alphamab

Nimotuzumab (803 words) [view diff] exact match in snippet view article find links to article

and Ukraine; for nasopharyngeal cancer in China. It has been granted orphan drug status for glioma in USA and for glioma and pancreatic cancer in Europe

Azasetron (410 words) [view diff] exact match in snippet view article find links to article

SENS-401 for Platinum-Induced Ototoxicity Sensorion has received the FDAs orphan drug designation for SENS-401 for the prevention of platinum-induced ototoxicity

YM Biosciences (515 words) [view diff] case mismatch in snippet view article find links to article

targeting the epidermal growth factor receptor. YM BioSciences received Orphan Drug Designation from the FDA for Nimotuzumab in 2004 and received a number

YM Biosciences (515 words) [view diff] case mismatch in snippet view article find links to article

targeting the epidermal growth factor receptor. YM BioSciences received Orphan Drug Designation from the FDA for Nimotuzumab in 2004 and received a number

Leadiant Biosciences (1,996 words) [view diff] case mismatch in snippet view article find links to article

pharmaceutical company in the world and assignee in the United States of an Orphan Drug Designation, subsequently receiving another seven. With the discovery

Precigen (296 words) [view diff] exact match in snippet view article find links to article

Precigen, with a focus on human gene therapy. Precigen went on to win orphan drug status from the FDA for a CAR-T based therapy (investigational drug name

Occipital horn syndrome (1,397 words) [view diff] exact match in snippet view article find links to article

AAV-ATP7A is still in pre-clinical stage, although it has received orphan drug designation from the FDA.[citation needed] Cutis laxa List of cutaneous

Neurovax (139 words) [view diff] case mismatch in snippet view article find links to article

Designation on February 6, 2014 & FDA Fast Track Designation for SPMS. "Search Orphan Drug Designations and Approvals". Retrieved 13 January 2015. "Neurovax". Retrieved

Sense (molecular biology) (2,444 words) [view diff] exact match in snippet view article

healio. 1 October 1998. Retrieved 18 September 2020. "FDA approves orphan drug for inherited cholesterol disorder". Drug Topics. 30 January 2013. Retrieved

Amylin Pharmaceuticals (1,234 words) [view diff] case mismatch in snippet view article find links to article

plant during sale". daytondailynews. Retrieved 2017-05-25. Lipodystrophy Orphan Drug Program. Amylin Pharmaceuticals. U.S. Food and Drug Administration (25

HealthCap (584 words) [view diff] exact match in snippet view article find links to article

remodeling and fracture healing developed by BoneSupport AB. WTX101, an orphan drug currently in development by Wilson Therapeutics for the treatment of

Orthopoxvirus (1,823 words) [view diff] exact match in snippet view article find links to article

orthopoxviruses in vitro and among test animals. Tecovirimat has been granted orphan drug status by the Food and Drug Administration (FDA) and is currently under

Histone deacetylase inhibitor (2,960 words) [view diff] exact match in snippet view article find links to article

latently infected T-cells in vitro and ex vivo. Givinostat (ITF2357) is an orphan drug for treatment of polycythemia vera (PV), essential thrombocythemia (ET)

Protein kinase inhibitor (742 words) [view diff] case mismatch in snippet view article find links to article

molecule 2006 Entrectinib TrkA/TrkB/TrkC/ROS1/ALK Ignyta Small molecule Orphan Drug Designations (Neuroblastoma 12/14, Colorectal cancer, NSCLC, both 2/15)

Protein kinase inhibitor (742 words) [view diff] case mismatch in snippet view article find links to article

molecule 2006 Entrectinib TrkA/TrkB/TrkC/ROS1/ALK Ignyta Small molecule Orphan Drug Designations (Neuroblastoma 12/14, Colorectal cancer, NSCLC, both 2/15)

Chronic myelogenous leukemia (3,797 words) [view diff] exact match in snippet view article find links to article

Administration. October 26, 2012. Retrieved October 27, 2012. "FDA approves new orphan drug for chronic myelogenous leukemia" (Press release). US Food and Drug Administration

Rare Disease Day (1,103 words) [view diff] case mismatch in snippet view article find links to article

"rare day," and 2008 was the 25th anniversary of the passing of the Orphan Drug Act in the United States. Individuals observing Rare Disease Day took

Choroideremia (3,087 words) [view diff] case mismatch in snippet view article find links to article

April 2015. Cory MacDonald, 2015, "Nightstar Receives U.S. and European Orphan Drug Designation for Gene Therapy to Treat Choroideremia," Choroideremia Research

Paclitaxel trevatide (1,252 words) [view diff] exact match in snippet view article find links to article

2009 and updated in June 2010. In 2014, paclitaxel trevatide received orphan drug designation from FDA for the treatment of glioblastoma multiform As of

Endoglin (2,839 words) [view diff] case mismatch in snippet view article find links to article

585–603. doi:10.1158/1541-7786.mcr-06-0408. PMID 17579118. "TRC105 Gets Orphan Drug Status for Soft-Tissue Sarcoma. Feb 2016". cancernetwork.com. 2016-02-04

Allan–Herndon–Dudley syndrome (1,554 words) [view diff] exact match in snippet view article find links to article

Allan–Herndon–Dudley syndrome.[citation needed] In May 2013, the US FDA granted Orphan drug status to Diiodothyropropionic acid (DITPA) in the treatment of MCT8

Instituto Bioclon (313 words) [view diff] exact match in snippet view article find links to article

Bioclon Institute is the only Mexican company that has obtained an "orphan drug" status from the Food and Drug Administration (FDA) of the United States

Heavy isotope diet (519 words) [view diff] exact match in snippet view article find links to article

Friedreich’s ataxia and infantile neuroaxonal dystrophy. FDA has granted it an orphan drug designation and it passed the Phase I/II clinical trials (as of 2018)

Emergent BioSolutions (7,277 words) [view diff] exact match in snippet view article find links to article

United Kingdom, Italy and the Netherlands. The FDA gave BioThrax an "orphan drug" designation in April 2014. The FDA gives that status to drugs that are

Amicus Therapeutics (816 words) [view diff] case mismatch in snippet view article find links to article

Cory (2014). "Chapter 4. Treating Rare Diseases: Business Model for Orphan Drug Development". In Pryde, David C.; Palmer, Michael J. (eds.). Orphan Drugs

Mipomersen (786 words) [view diff] exact match in snippet view article find links to article

Disease". The New York Times. Retrieved 31 January 2013. "FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder". U.S. Food and Drug

Emergent BioSolutions (7,277 words) [view diff] exact match in snippet view article find links to article

United Kingdom, Italy and the Netherlands. The FDA gave BioThrax an "orphan drug" designation in April 2014. The FDA gives that status to drugs that are

List of drugs: E–El (449 words) [view diff] exact match in snippet view article find links to article

Eldecort Eldepryl eledoisin (INN) Elestat elesclomol (investigational drug/orphan drug) eletriptan (INN) Elevidys Elfabrio elfazepam (INN) elgodipine (INN)

Mipomersen (786 words) [view diff] exact match in snippet view article find links to article

Disease". The New York Times. Retrieved 31 January 2013. "FDA approves new orphan drug Kynamro to treat inherited cholesterol disorder". U.S. Food and Drug

C6H10N2O5 (64 words) [view diff] exact match in snippet view article find links to article

a zwitterionic organic chemical buffering agent Carglumic acid, an orphan drug marketed by Orphan Europe under the trade name Carbaglu This set index

Ricin (6,618 words) [view diff] exact match in snippet view article find links to article

issued a US patent for Rivax. The ricin vaccine candidate was granted orphan drug status in the US and the EEC and, as of 2019, was in clinical trials

Infectious Diseases Society of America (2,751 words) [view diff] case mismatch in snippet view article find links to article

smaller, shorter, and less expensive clinical trials, similar to the Orphan Drug Program. In addition to the 10 x '20 Initiative, IDSA supports legislative

Factor X (3,302 words) [view diff] exact match in snippet view article find links to article

original on October 21, 2015. Retrieved October 21, 2015. Until today's orphan drug approval, no specific coagulation factor replacement therapy was available

Delcath Systems (366 words) [view diff] case mismatch in snippet view article find links to article

University of Texas Medical Branch in Galveston, Texas "Melphalan granted FDA Orphan Drug Designation for treatment of cholangiocarcinoma", News-Medical.Net, 20

Federal Food, Drug, and Cosmetic Act (3,338 words) [view diff] case mismatch in snippet view article find links to article

(May 28, 1976) Infant Formula Act of 1980, PL 96–359 (October 26, 1980) Orphan Drug Act, PL 97–414 (January 4, 1983) Drug Price Competition and Patent Term

Gordon Binder (301 words) [view diff] case mismatch in snippet view article find links to article

Ltd (2015). Mr Henri Termeer: Chief Executive Officer, Former Genzyme. Orphan Drug Congress. Retrieved 27 September 2015. The MIT Corporation American Enterprise

Coramsine (933 words) [view diff] exact match in snippet view article find links to article

glycosides against cancer cells. During 2005 and 2006 Solbec was granted orphan drug designation for Coramsine by the U.S. Food and Drug Administration in

Axona (1,330 words) [view diff] case mismatch in snippet view article find links to article

classification, and was originally defined by Congress as part of the Orphan Drug Amendments of 1988 as "a food which is formulated to be consumed or administered

Neurolixis (1,998 words) [view diff] case mismatch in snippet view article find links to article

of Huntington's disease. F-15599 (also known as NLX-101) was awarded Orphan Drug Status by the United States Food and Drug Administration (FDA) in October

Solanum nigrum (3,791 words) [view diff] exact match in snippet view article find links to article

carbon C-22 at 110.5 and 112.5 ppm, respectively. US-FDA granted an orphan drug designation for uttroside B against hepatocellular carcinoma. Black nightshade

Small interfering RNA (6,760 words) [view diff] case mismatch in snippet view article find links to article

substantial improvement over any available therapy. It was also awarded Orphan Drug Designations given to those treatments that are intended to safely treat

Merck & Co. (9,284 words) [view diff] case mismatch in snippet view article find links to article

1016/S0016-5085(89)80061-7. PMID 2909442. "Shocker! Is Vioxx Coming Back... as an Orphan Drug?". www.medpagetoday.com. 2018-05-02. Berenson, Alex; Harris, Gardiner;

Behçet's disease (5,148 words) [view diff] case mismatch in snippet view article find links to article

controlled study". J Rheumatol. 32 (1): 98–105. PMID 15630733. "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 19 November

Remune (404 words) [view diff] case mismatch in snippet view article find links to article

-fda-has-accepted-remune-hivaids-vaccine-bla-for-review.html "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2015-05-19

Roseburia hominis (315 words) [view diff] case mismatch in snippet view article find links to article

Microbiology. 56 (Pt 10): 2437–2441. doi:10.1099/ijs.0.64098-0. PMID 17012576. "Orphan Drug Designations and Approvals". Food and Drug Administration. Staley, James

Maryland (18,571 words) [view diff] case mismatch in snippet view article find links to article

20, 2011. Retrieved August 19, 2011. "Emergent BioSolutions Receives Orphan Drug Designation for BioThrax for Post-Exposure Prophylaxis of Anthrax Disease"

Viloxazine (3,053 words) [view diff] case mismatch in snippet view article find links to article

Analogue-based Drug Discovery. John Wiley & Sons. ISBN 978352760749-5. "Orphan Drug Designations and Approvals: Viloxazine". U.S. Food and Drug Administration

Prescription Drug User Fee Act (3,146 words) [view diff] case mismatch in snippet view article find links to article

approval of Videx. AIDS groups fought for the reauthorization of the Orphan Drug Act and the passage of the Prescription Drug User Fee Act in 1992. The

Entolimod (1,044 words) [view diff] case mismatch in snippet view article find links to article

action of the compound. The FDA granted entolimod both Fast Track and Orphan Drug status for reducing the risk of death following a potentially lethal

Azurin (1,658 words) [view diff] exact match in snippet view article find links to article

tumors; subsequently, the USFDA approved the designation of p28 as an orphan drug for glioma. Azurin’s other domains may also exhibit strong anticancer

Chiesi Farmaceutici (1,115 words) [view diff] exact match in snippet view article find links to article

chiesi.com. Retrieved 2019-07-26. "Chiesi's Holoclar wins UK Prix Galien orphan drug award". PMLive. 2016-09-23. Retrieved 2019-07-26. McKee, Selina (2018-04-05)

Frederick Sachs (892 words) [view diff] exact match in snippet view article find links to article

Drug Administration designated the firm’s peptide, called GsMTx4, as an orphan drug for Duchenne muscular dystrophy. In 2012, Sachs and Harvey opened their

Enadenotucirev (372 words) [view diff] exact match in snippet view article find links to article

ISSN 2051-1426. PMC 6348630. PMID 30691536. "EMA grants positive opinion for orphan drug status for ovarian cancer oncolytic vaccine". PsiOxus Therapeutics. 2015-01-13

Mitzi Nagarkatti (1,573 words) [view diff] exact match in snippet view article find links to article

autoimmune hepatitis was the basis for FDA to approve its use as an orphan drug to treat this disorder. Nagarkatti's laboratory has also studied the

TK cell therapy (487 words) [view diff] case mismatch in snippet view article find links to article

expressing TK in patients with graft-versus-host disease. TK has been granted Orphan Drug designation both in the EU and the United States "TK008: Randomized Phase

Burzynski Clinic (6,930 words) [view diff] exact match in snippet view article find links to article

2011 Dr. Stanislaw Burzynski, antineoplastons, and the selling of an orphan drug as a cancer cure Quote: " it’s not the concept of "personalized gene-targeted

Tolerx (623 words) [view diff] case mismatch in snippet view article find links to article

and commercialization of otelixizumab. Otelixizumab has been granted Orphan Drug Status by the U.S. Food and Drug Administration. Additionally, in collaboration

Non-24-hour sleep–wake disorder (6,317 words) [view diff] exact match in snippet view article find links to article

clinical trial in the United States since 2002 and the FDA granted it orphan drug designation in May 2004, for use as a treatment for circadian rhythm

Neglected tropical diseases (14,950 words) [view diff] exact match in snippet view article find links to article

http://www.mectizan.org/ Villa S, Compagni A, Reich MR. (January–March 2009) "Orphan drug legislation: lessons for neglected tropical diseases". Int J Health Plann

Stephen Neidle (1,423 words) [view diff] case mismatch in snippet view article find links to article

USA Food and Drug Administration approved the lead compound QN-302 for Orphan Drug designation in pancreatic cancer. In July 2023 Qualigen Therapeutics

Paul A. Hopper (1,281 words) [view diff] case mismatch in snippet view article find links to article

Theranostics, Radiopharm (9 May 2023). "Radiopharm Theranostics Receives FDA Orphan Drug Designation for Trivehexin (RAD 301) in Pancreatic Cancer". GlobeNewswire

Medac (552 words) [view diff] exact match in snippet view article find links to article

preparation Treosulfan, which Medac manufactured itself, was classified as an orphan drug. The pharmaceuticals and diagnostics produced by Medac are delivered

TREAT-NMD (605 words) [view diff] exact match in snippet view article find links to article

committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development". Orphanet Journal of Rare Diseases. 10 (1): 49. doi:10

Societal and cultural aspects of Tourette syndrome (6,478 words) [view diff] case mismatch in snippet view article find links to article

Tourette's as an organic disorder, but also helped get the then stalled 'Orphan Drug Bill' passed ... into legislation". The entertainment industry has been

Henry Lowe (1,985 words) [view diff] exact match in snippet view article find links to article

leukaemia. On July 12, 2017, Lowe announced Chrysoeriol had been given orphan drug status by the FDA Lowe, working with various academics, has filed for

Consumer health laws (1,148 words) [view diff] case mismatch in snippet view article find links to article

ensure the nutritional content and safety of commercial baby foods. Orphan Drug Act (1983) – Facilitated the development of new drugs for more than 5000

Alan B. Scott (2,339 words) [view diff] case mismatch in snippet view article find links to article

thousands of patients collected by 240 investigators, under the 1983 US Orphan Drug Act, Scott obtained FDA approval in 1989 to market Oculinum for clinical