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In the past 5 years, the FDA has approved a total of 5 DMD drugs, all of which will enter China through different routes:
In the past 5 years, the FDA has approved a total of 5 DMD drugs, all of which will enter China through different routes:
Amondys 45/Exondys 53/Vyondys 51
Amondys 45/Exondys 53/Vyondys 51In September 2016, the FDA approved Exondys of Sarepta Therapeutics, the first Duchenne muscular dystrophy (DMD) drug
51 (Eteplirsen), used to treat approximately 13% of DMD patients
with identified exon 51 mutations.
Vyondys 53 (golodirsen) and Amondys of Sarepta Therapeutics
45 (casimersen) was approved by the FDA in December 2019 and February 2021 for the treatment of
patients with exon 45 jump (about 8%) and exon 53 jump (about 8%) DMD, respectively.
Amondys 45/Exondys 53/Vyondys
All 51 are antisense oligonucleotide drugs that use Sarepta's proprietary phosphodiamide morpholiline oligomeric (PMO) chemistry and exon-skipping technology to skip exons 45/53/51 of the DMD gene, respectively
.
Three drugs were designed to bind dystrophin pre
Exon 45/53/51 of the mRNA, thereby excluding or "skipping" exon 45/53/51 during mRNA processing in DMD patients with genetic mutations suitable for skipping exons 45/53
/51.
Exon skipping is done to allow the production of an internally truncated dystrophin
.
Recently, with the support of the licensed medical policy of Boao Lecheng International Medical Tourism Pilot Zone, Boao Lecheng Weijian Rare Disease Clinical Medical Center successfully introduced EXONDYS 51 and VYONDYS
53、AMONDYS
45
。 For DMD patients who meet the requirements, these three drugs are accessible to patients in Lecheng, providing one more choice for patients and families, and also providing a formal path
for patients to obtain good treatment.
Viltepso（viltolarsen）
Viltepso（viltolarsen）In August 2020, the FDA approved the launch of a second drug targeting the exon 53 jumping DMD gene mutation, NS, a subsidiary of Nippon Shinyaku
Viltepso (viltolarsen)
developed by Pharma.
On January 20 this year, the phase II clinical trial of Viltolarsen, the first international multi-center 53 jumping drug in Hunan Children's Hospital, was launched
.
Viltolarsen is expected to be declared for listing
through the State Food and Drug Administration in the future.
Emflaza（deflazacort）
Emflaza（deflazacort）In February 2017, the FDA approved PTC
Therapeutics drug Embraza (deflazacort) is used for the treatment
of DMD patients 5 years of age and older.
Emflaza is the world's first corticosteroid drug
approved for the treatment of DMD.
Compared with the other 4 antisense oligonucleotide drugs, Emflaza is more widely used in the population and is suitable for all types of DMD patients
with gene mutations.
It is reported that Emflaza, developed by Faes.
Farma in Spain, was bought by Marathon in the United States in 2003 and later sold to PTC
.
Then, in 2017, PTC used Faes.
Farma's pharmaceutical data to obtain FDA approval
.
In 2018, Macao Wuzhou Pharmaceutical signed a license agreement with Faes.
Farma of Spain, introducing Defal, which is the same drug name as Emflaza, to China, and was approved for marketing in Macao in 2019, and then began to sell
to the mainland at a low price with the policy of the "Guangdong-Hong Kong-Macao Greater Bay Area Drug and Medical Device Regulatory Innovation and Development Work Plan".
It is understood that in addition to obtaining marketing authorization from the China Food and Drug Administration, the introduction policy of the Guangdong-Hong Kong-Macao Greater Bay Area is currently the only way
to obtain formal prescription drugs abroad.
Drugs introduced through the GBA need to submit the required documents for registration application to the Hong Kong and Macao Food and Drug Administration, including production formula, product specifications, laboratory report and manufacturer's license, etc.
, for approval by the Food and Drug Administration to ensure that the drugs sold are safe, effective and of high quality
.
