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My Thank You Email To The FDA, Which Applies To The Entire #Myeloma Care Team!

8/20/2020

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FDA, Thank You Again For giving #Myeloma Patients A new LAST CHANCE AT LIFE!

Thank you for the approval of belantamab mafodotin for late stage myeloma treatment. The myeloma patient community believes the cooperative efforts instituted in the FDA orphan drug program has given myeloma patients so many added years of life. When I was diagnosed 13 years ago life expectancy was just 33 month. It is now TWICE as long! The following is the reason we have belief we have a future.

Myeloma patients should feel blessed to know they have had 10 new drugs and drug classes approved in the last 16 years all of which all had two FDA designations. In addition, Myeloma (an orphan disease) has 7 drugs which all had two FDA designations, but have yet to be approved. These drugs include the following:

Drug                             Company                 Designations      Description

CLR131                         Cellectar              OD&FT           Radiotherapeutic phospholipid

JNJ-4528 Janssen OD&BT CAR-T with two BCMS-targeting
Single domain antibodies
CT053 CARsgen OD, RMAT* A fully human anti-BCMA CAR--T

bb2121                          Celgene                       OD&BT          BCMA chimeric antigen receptor
CAR T
Galinpepimut-S              Sellas OD&FT Immunotherapy vaccine to elicit
strong response against WT1
P-BCMA-101 Poseida OD,FT&BT Autologous chimeric antigen
   receptor(CAR) T-cell therapy
Melflufen Oncopeptides OD&AA Activated by aminopeptidases,
overexpressed in myeloma
So how have we fared with this list?

AMG420 - has dropped from the list in favor of a kinder gentler similar drug called AMG701, however AMG701 has only one FDA designation.

All of the other drugs on the list are progressing very well, with  bb2121 expected to be approved in the next several months.

Best Regards/ Gary Petersen [email protected]

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Orphan Disease's Biggest Lost Opportunity! The FDA and NCI Need Advocate Input!

8/17/2020

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If Orphan cancers were a disease category by itself, it would be one of the top 5 largest killers in the USA just behind Heart Disease, Cancer, and #COVID19. I have myeloma and had the opportunity to go to ASH 2019, and it happened to be one of the best opportunities to see the future of blood cancer treatments. For many of the meetings you will see some of the most significant developments in cancer treatment like CAR T, ADC(Anitbody Drug Conjugates), AWC(Antibody Warehead Conjugates), BiTE’s, etc. and they are held in rooms which can seat hundreds or sometimes thousands of people.

However, the one thing all of the drugs under development have in common is they all must obtain FDA approval. The FDA had two meetings scheduled. It was for this reason I wanted to make sure I attended the meeting titled “FDA Dialogue with Patient Representatives and Advocates”. Myeloma clinicians and advocates had just gone through a very difficult approval process for Selinexor, and I wanted to find out what I could learn at this meeting. I almost missed the meeting because most are in large halls, and this meeting was in a meeting room, so I kept walking past it thinking this little room could not be the venue for a meeting as important as it seemed to me. As you can see in the following image the advocates meeting was in OfficeW206, and the other FDA meeting and most meetings in a hall the size of the grand Hall D.

At this meeting the FDA was well represented with 38 in attendance, and they seemed to outnumber the advocates by a factor of two to one. I was frankly shocked at the poor turnout of advocates, in that these FDA officials held the final judgment on approval or rejection.

Myeloma had two representatives so we were fairly well represented, and we were also quite willing to participate in the discussion. We had the opportunity to learn about the programs for orphan diseases which are designed to Increase the number of orphan diseases which have FDA approved drugs. Of the 7000 orphan diseases this FDA program had provided just over 500 new drugs. and this is just since the first Orphan Drug Act was passed in 1983. Prior to this FDA program few, if any, new drugs were ever approved for orphan diseases with less than 50 FDA approved orphan disease drugs available. The average orphan drug has annual sales of $350 million dollars each year, so most large pharmaceutical companies find this level of added sales is not worth their effort and the $2 billion dollars to bring them to market. The FDA orphan drug program has developed a designation which drug companions can apply, and this provides tax benefits and help in getting through the FDA approval process. It is because of these incentives that many smaller companies have become the engine of orphan drug development. The FDA has added other designations like Fast Track, Priority Review, and Breakthrough designations which help to expedite drugs through development. In addition, the National Cancer Institute, American Cancer Society, MMRF, LLS, Academic Institutions, and many others provide research grants to help fund Orphan Drug development. All of these organizations and the researchers and disease specialists are our allies in this life and death struggle. We can only thank these groups for their efforts to help to promote orphan drug development, without which there would be NO PROGRESS!

We were able to provide inputs to the group, praised them for final approval of Selinexor, asked for more representation specific to the disease to be at the the Oncology Drug Advisory Committee meetings to discuss the FDA concerns, and to recognize this is a last chance at life for late stage patients. For example if there were just one more myeloma specialist (or a total of two) of the 13 members on the ODAC(oncology drugs advisory committee), Selinexor would have been approved months earlier.

Small Biotech companies are at a significant disadvantage in the universe of drug companies. They are small and find it hard to fund themselves as they work through a 8 to 10 year FDA approval process. They seldom profit during these initial development years and find it impossible to borrow money, so they must depend on the capital markets for funding. As I had written before, the SEC has few tools to protect these baby biotechs from Capital Vultures(CLICK HERE for the article) who manipulate the stock, an illegal tactic driving the stock price down. This makes it even more difficult to fund their trials and these baby biotechs are forced into bankruptcy. I would love to see the SEC with the enforcement power to put these animals in jail, as well as the government provide a guaranteed loan program as another funding avenue, and prevent the use of viscous short sale attacks designed to profit from the drastic decline in a vulnerable biotech stock price. Because of these disadvantages we need all the allies we can get and must have more protections for small biotech firms.

One way as advocates and disease representatives to champion your orphan disease would be to attend any of these FDA/Advocate programs. Or send this article to your Senate and House representatives (CLICK HERE for contact information for your senators and representatives), @secazar, and @SteveFDA .

Good Luck and may God Bless your family's cancer journey. You can see more information on myeloma at my site www.myelomasurvival.com.

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My Thank You Email To The FDA, Which Applies To The Entire #Myeloma Care Team!

Orphan Disease's Biggest Lost Opportunity! The FDA and NCI Need Advocate Input!

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