PTC124 for Nonsense Mutations

M

MargaritaChic

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>

PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote></div>


I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?

My daughter has a nonsense mutation. I have been watching news reports of this drug.
 
M

MargaritaChic

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>

PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote></div>


I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?

My daughter has a nonsense mutation. I have been watching news reports of this drug.
 
M

MargaritaChic

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>

PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote></div>


I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?

My daughter has a nonsense mutation. I have been watching news reports of this drug.
 
M

MargaritaChic

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>

PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote>


I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?

My daughter has a nonsense mutation. I have been watching news reports of this drug.
 
M

MargaritaChic

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>
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<br />PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote>
<br />
<br />
<br />I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?
<br />
<br />My daughter has a nonsense mutation. I have been watching news reports of this drug.
<br />
<br />
 
M

MargaritaChic

New member
<b>Orphan drug Status:</b>
In the U.S, an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ("orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States or low prevalence is taken as prevalence of less than 5 per 10,000 in the community. This has been adopted as a subclause of the Food and Drug Administration (FDA) regulations. The granting of orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive/un-profitable to develop under normal circumstances. Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity (a "monopoly") on that drug for an extended time (seven years post-approval). The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research.
 
M

MargaritaChic

New member
<b>Orphan drug Status:</b>
In the U.S, an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ("orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States or low prevalence is taken as prevalence of less than 5 per 10,000 in the community. This has been adopted as a subclause of the Food and Drug Administration (FDA) regulations. The granting of orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive/un-profitable to develop under normal circumstances. Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity (a "monopoly") on that drug for an extended time (seven years post-approval). The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research.
 
M

MargaritaChic

New member
<b>Orphan drug Status:</b>
In the U.S, an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ("orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States or low prevalence is taken as prevalence of less than 5 per 10,000 in the community. This has been adopted as a subclause of the Food and Drug Administration (FDA) regulations. The granting of orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive/un-profitable to develop under normal circumstances. Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity (a "monopoly") on that drug for an extended time (seven years post-approval). The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research.
 
M

MargaritaChic

New member
<b>Orphan drug Status:</b>
In the U.S, an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ("orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States or low prevalence is taken as prevalence of less than 5 per 10,000 in the community. This has been adopted as a subclause of the Food and Drug Administration (FDA) regulations. The granting of orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive/un-profitable to develop under normal circumstances. Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity (a "monopoly") on that drug for an extended time (seven years post-approval). The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research.
 
M

MargaritaChic

New member
<b>Orphan drug Status:</b>
<br />In the U.S, an orphan drug is any drug developed under the Orphan Drug Act of January 1983 ("ODA"), a federal law concerning rare diseases ("orphan diseases"), defined as diseases affecting fewer than 200,000 people in the United States or low prevalence is taken as prevalence of less than 5 per 10,000 in the community. This has been adopted as a subclause of the Food and Drug Administration (FDA) regulations. The granting of orphan drug status is designed to encourage the development of drugs which are necessary but would be prohibitively expensive/un-profitable to develop under normal circumstances. Because medical research and development of drugs to treat such diseases is financially disadvantageous, companies that do so are rewarded with tax reductions and marketing exclusivity (a "monopoly") on that drug for an extended time (seven years post-approval). The concept behind the ODA is that the longer period of exclusivity will encourage more companies to invest money in research.
 
L

lflatford

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>MargaritaChic</b></i>

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>



PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote></div>

Because only 70,000 people worldwide 35,000 in the US have CF it is considered an orphan disease based on the number of people who have the disease.

Chuck





I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?



My daughter has a nonsense mutation. I have been watching news reports of this drug.</end quote></div>
 
L

lflatford

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>MargaritaChic</b></i>

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>



PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote></div>

Because only 70,000 people worldwide 35,000 in the US have CF it is considered an orphan disease based on the number of people who have the disease.

Chuck





I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?



My daughter has a nonsense mutation. I have been watching news reports of this drug.</end quote></div>
 
L

lflatford

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>MargaritaChic</b></i>

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>



PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote></div>

Because only 70,000 people worldwide 35,000 in the US have CF it is considered an orphan disease based on the number of people who have the disease.

Chuck





I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?



My daughter has a nonsense mutation. I have been watching news reports of this drug.</end quote></div>
 
L

lflatford

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>MargaritaChic</b></i>

<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>



PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote>

Because only 70,000 people worldwide 35,000 in the US have CF it is considered an orphan disease based on the number of people who have the disease.

Chuck





I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?



My daughter has a nonsense mutation. I have been watching news reports of this drug.</end quote>
 
L

lflatford

New member
<div class="FTQUOTE"><begin quote><i>Originally posted by: <b>MargaritaChic</b></i>
<br />
<br /><div class="FTQUOTE"><begin quote><i>Originally posted by: <b>kitomd21</b></i>
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<br />
<br />
<br />PTC124 has been granted orphan drug status by the FDA and the European Commission for the treatment of CF and DMD due to nonsense mutations. The FDA has also granted PTC124 Subpart E designation for expedited development, evaluation, and marketing.</end quote>
<br />
<br />Because only 70,000 people worldwide 35,000 in the US have CF it is considered an orphan disease based on the number of people who have the disease.
<br />
<br />Chuck
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<br />
<br />
<br />I do not understand the paragraph quoted above. What is orphan drug status and what is Subpart E designation?
<br />
<br />
<br />
<br />My daughter has a nonsense mutation. I have been watching news reports of this drug.</end quote>
<br />
<br />
 
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