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Orphan drugs
1. ORPHAN DRUGS
After covid-19 infection
Prepared by –
Mujeeb Mahamadhusen Shaikh
Faculty –
M.Pharmacy (PQA)
Under guidance
Dr. Anwar Shaikhsir
HOD of PQA Department
AllanaCollage Of Pharmacy, Camp Pune 01
2. Orphan Drugs- Drugsusedto treata rare disease are called“OrphanDrugs“
Where there are lessthan2 lakhscasesper year,or where salesof the drugdo not ensure adequate
returns,the drugsare classifiedasOrphanDrugs
List of Orphan drugs : (SADSO FAR)
Somatotropin – Growthhormone secretedbypituitarygland.
Ancrod (Viprinex) –Defibrinogenatingagent(Anticoagulanteffect) derivedfromMalayanpit
viper
Digoxin– Cardiacglycoside usedintreatmentof mildtomoderate heartfailure.
LiOthyronine –It isthyroidhormone usedtotreathypothyroidismandmyxedemacoma.
Formepizole(4-methylpyrazole) –usedtotreat methanol andethylene glycolpoisoning.
AmphotericinB– Antifungal agentusedtoseriousfungalinfectionandleishmaniasis.
Rifabutin–Antibioticused totreatTB and preventMycobacteriumaviumcomplex.
After covid19 serious side effectMUCORMICSIS alsocalledBLACKFUNGUS
Mucormycosis (previously called zygomycosis) is a serious but rare fungal infection caused by a
group of molds called mucormycetes. These molds live throughout the environment.
Mucormycosis mainly affects people who have health problems or take medicines that lower the
body’s ability to fight germs and sickness. It most commonly affects the sinuses or the lungs
after inhaling fungal spores from the air. It can also occur on the skin after a cut, burn, or other
type of skin injury.
TYPE of MUCORMICOSIS
Rhinocerebral (sinus and brain) mucormycosis itaffectpeople with uncontrolled
diabetes and in people who have had a kidney transplant.
Pulmonary (lung) mucormycosis – common infection in peoplewith cancer and on
organ transplant or stem cell transplant.
Gastrointestinal mucormycosis – common in young children than adults, especially
premature and low birth weight infants less than 1 month of age, who have had
antibiotics, surgery, or medications that lower the body’s ability to fight germs and
sickness.
Cutaneous (skin) mucormycosis: occurs after the fungi enter the body through a break
in the skin (for example, after surgery, a burn, or other type of skin trauma). This is the
3. most common form of mucormycosis among people who do not have weakened
immune systems.
Disseminated mucormycosis occurs when the infection spreads through the bloodstream
to affect another part of the body. The infection most commonly affects the brain, but also
can affect other organs such as the spleen, heart, and skin.
Role of AmphotericinB inBlack fungaus
AMphotericinBclassifiedasbindtofungal cell membrane ergosterol.
One of the mainusesistreatingwide range of systemicfungal infections.Due toitslessside effect.
IT is oftenreservedforseverinfectionsincriticallyillorimmunosupressedpatients.
It considerdfirstline therapyforintensive Mucormicosisinfection,cryptococcal meningitisandcertain
aspergiliusandcandidal infections.
It ishighlyeffective drugsforover50 yearsin large part because ithaslow incidence of drugresistance
due to it require sacrificesonpartof pathogenthatmake ie susceptibletohostenvironmentandtoo
weakto cause infection.
How do you get mucormycosis?
Mucorales are considered opportunistic fungi,meaning they usuallyinfect people with an impaired immune system,
or with damaged tissue.Use ofdrugs which suppress the immune system such as corticosteroids can lead to
impaired immune function,as can a range of other immunocompromising conditions,like cancer or transplants.
Damaged tissue can occur after trauma or surgery.
There are three ways humans can contractmucormycosis — by inhaling spores,byswallowing spores in food or
medicines,or when spores contaminate wounds.
Inhalation is mostcommon.We actually breathe in the spores ofmanyfungi every day. But our immune system and
healthy lungs generallyprevent them from causing an infection.
When the lungs are damaged and the immune system is suppressed,such as is the case in patients with severe
COVID, these spores can grow in our airways or sinuses and invade our bodies’ tissue.
People with diabetes and obesitytend to develop more severe COVID infections.This means they’re more likelyto
receive corticosteroids,which are frequently used to treat COVID-19. But the corticosteroids — along with their
diabetes — increase the risk of mucormycosis.
Meanwhile, COVID itselfcan damage airwaytissue and blood vessels,which could also increase susceptibilityto
fungal infection.
So damage to tissue and blood vessels from COVID infection,treatmentwith corticosteroids,high background rates
of diabetes in the population mostseverelyaffected by COVID, and,importantly, more widespread exposure to the
fungus in the environmentare all likely to be playing a part in the situation we’re seeing with mucormycosis in India
4. About AmphotericinB
- ObtainedfromstreptomycesNodosus
- Amphotericinnature
- ROA – usuallyI.V.(slowinfusiononly)
- BA – 100%
- Metabolism–Kidney
- M.F- C47H73NO17
- Molar Mass – 924.091g/mol-1
- M.P – 170 degree cel.
- Structure
Mechanism Of Action -
It binds with ergosterol is a componentoffungal cell membrane,
Forming pores thatcause rapid leakage ofn monovalent ions (K+,Na+ & Cl-) and subsequenr fungal cell death.
.
References-
https://en.wikipedia.org/wiki/Amphotericin_B
https://health.economictimes.indiatimes.com/news/industry/mucormycosis-an-emerging-challenge-in-covid-
19-management/82507750
https://www.cdc.gov/fungal/diseases/mucormycosis/definition.html
Lewis RE,Kontoyiannis DP- Epidemiology& Treatmentof mucormycosisexternal icon.Futeure Microbiol
2013
https://go.drugbank.com/drugs/DB00681
https://theconversation.com/what-is-mucormycosis-the-fungal-infection-affecting-covid-patients-in-india-
160707