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Saniona meddelade igår att FDA har beviljat särläkemedelsklassning till läkemedelskandidaten Tesomet för behandling av Prader-Willis syndrom (PWS). BioStock kontaktade Rudolf Baumgartner, MD, Chief Medical Officer och Head of Clinical Development på Saniona, för att få hans syn på denna viktiga milstolpe och på vilka sätt denna nya klassning kommer att hjälpa Saniona framåt.

I Europa definieras en sjukdom som sällsynt när den drabbar färre än 1 av 2000 personer, medan man i USA, baserat på ett beslut av den amerikanska kongressen i Orphan Drug Act of 1983, är ett tillstånd som drabbar färre än 200 000 personer.

Sammantaget är sällsynta sjukdomar dock inte särskilt sällsynta. Enligt en vetenskaplig artikel som publicerades i European Journal of Human Genetics år 2019, beräknas antalet människor världen över som lever med någon av de cirka 7000 identifiera sällsynta sjukdomarna vara 300 miljoner, vilket motsvarar nästan hela USAs befolkning.

Detta återspeglas också av marknaden för särläkemedel där den globala försäljningen förväntas nå 413 miljarder USD år 2030.

»Having orphan drug designation has been proven to help aid in the approval of orphan drugs: in the 16 years prior to the Orphan Drug Act going into effect, the FDA approved only 34 drugs for rare diseases. Since the Act went into effect in 1983, the FDA has approved more than 600 orphan products!« – Rudolf Baumgartner, Chief Medical Officer and Head of Clinical Development Saniona

Prader-Willis syndrom

Antalet patienter som lever med Prader-Willis syndrom (PWS) uppskattas vara mellan 11 000 och 34 000 i USA och mellan 17 000 och 50 000 i Europa, vilket kvalificerar PWS som en sällsynt sjukdom både i USA och i Europa.

PWS är den vanligaste genetiska orsaken till livshotande övervikt och orsakas av en radering eller förlust av funktioner av ett kluster av gener på kromosom 15, vilket leder till dysfunktionell signalering i hjärnans aptit/mättnadscentrum (hypotalamus). Patienterna kännetecknas av låg muskelmassa och tidigt debuterande barnfetma, omättlig hunger, extrem fetma och utvecklingsstörning. De lider vanligen av en konstant och extrem aptit oavsett hur mycket de äter. Som ett resultat av detta dör många av patienterna i ung ålder.

Det finns inga läkemedel som är godkända specifikt för den omättliga aptiten (hyperfagi) som är associerad med PWS, och det finns inget botemedel mot sjukdomen. Behandlingen beror på symtom och inkluderar ofta hormonersättning. Om fetma kan undvikas och komplikationer hanteras väl, kan livslängden för dessa individer förbättras.

CMO kommenterar Orphan Drug Designation från FDA för Tesomet i PWS

Igår tillkännagav bioteknikföretaget Saniona att bolaget har erhållit Orphan Drug Designation (ODD) för Tesomet i Prader-Willis syndrom från FDA i USA. BioStock kontaktade Rudolf Baumgartner, MD, Chief Medical Officer och Head of Clinical Development på Saniona, som förklarar på vilka sätt särläkemedelsstatusen kommer att gynna Saniona på vägen mot nästa kliniska steg, en fas IIb-studie i PWS.

Rudolf Baumgartner, CMO and Head of Clinical Development Saniona

Rudolf Baumgartner, to start out with, ODD is by all definitions an important milestone in any biotech company, Saniona is no exception. In what ways will this special status from the FDA help Saniona going forward?

– Orphan Drug designation is granted by the Office of Orphan Products Development (OOPD) and is typically given to drugs or biologics intended for the treatment, diagnosis or prevention of a rare disease. And, as we know, a rare disease is defined as afflicting less than 200,0000 US patients.

– This designation has many benefits to Saniona and has been created to incentivize development of treatments in these very difficult areas. So, that ultimately, patients benefit. Some of the benefits include participation in the Orphan Products Grants Program which can provide funding for clinical research, tax credits, and waiver of certain FDA fees.

– Additionally, although it is not mentioned as much, one of the most important benefits of orphan drug designation, in my opinion, is the opportunity for Saniona’s clinical team to get additional agency advice and support, such as protocol assistance. This assistance is invaluable as it helps the Saniona team to better align the clinical development program with the agency’s perspective of what data needs to be created to allow the Division to make an informed decision on the eventual approvability of the treatment.

– Having orphan drug designation has been proven to help aid in the approval of orphan drugs: in the 16 years prior to the Orphan Drug Act going into effect, the FDA approved only 34 drugs for rare diseases. Since the Act went into effect in 1983, the FDA has approved more than 600 orphan products!

– Finally, orphan drugs that get FDA approval may be eligible for seven years of market exclusivity in the U.S., which can be a very valuable benefit.

You are currently planning to initiate a Phase IIb study with Tesomet in PWS during the first half of 2021. What work remains to be done before it can be initiated?

– The Saniona team has been working diligently to prepare to initiate the Phase 2b clinical trial of Tesomet in PWS. We have opened an Investigational New Drug (IND) filing with the FDA, selected the clinical research organization (CRO) that will support the clinical trial execution. We are in the process of assessing and selecting clinical trial sites in the U.S. and globally. We have also selected the contract manufacturer to produce Tesomet for Phase 2b and Phase 3 clinical trials The contract manufacturer is actively working on clinical production.

– Additionally, we have initiated multiple partnerships with the PWS advocacy community to ensure caregiver and patient feedback is incorporated into the trial designs and to provide the community with education on the upcoming clinical trials. We now need to continue our clinical site selection and manufacturing preparation processes. We will continue to work closely with the FDA on finalizing the design of the Phase 2b trial. We look forward to initiating the study in the first half of this year.

Saniona is also evaluating Tesomet for the treatment of hypothalamic obesity (HO), which is a very rare disease. You are currently planning to begin a phase IIb trial in this indication, also in the first half of this year. Are you hopeful that the ODD in PWS from the FDA could lead to yet another ODD in HO, given the similarities in these two rare diseases?

– Both PWS and HO are rare diseases with significant unmet need: there are no medicines approved for HO, and there are no medicines approved for the insatiable hunger/hyperphagia that characterizes PWS. In PWS, however, there are other pharmaceutical companies who are also developing potential treatments, and some of them had also applied for, and received, Orphan Drug Designation. Thus, a precedent exists in PWS.

– HO is different because there has not been a lot of research or pharmaceutical industry activity in trying to develop treatments for HO, so the FDA has never granted an orphan drug designation in HO. There is no precedent here; Saniona is attempting to pioneer a new trail. We have filed for orphan drug designation in HO but given the lack of precedent and the lack of visibility around this rare disease, we anticipate this process may require additional time.

– Even so, we see significant unmet need in the HO community also: it is estimated that between 10,000 and 25,000 people in the U.S. and between 16,000 and 40,000 people in Europe suffer from HO, and they experience rapid weight gain, hyperphagia, and many other symptoms that can decrease their quality of life and lead to significant morbidity and mortality. Thus, we continue to be dedicated to advancing Tesomet for HO as well as for PWS.

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